An International Crisis and Local Crisis and Epidemic in Our Community: New Models of Prevention, Treatment, and Recovery

COMMUNITY DAY PROGRAM AGENDA

Steven Huberman, PhDDean, Touro College Graduate School of Social Work

Alan Kadish, MDPresident, Touro College and University System

Arlene Gonzalez-Sanchez, MS, LMSWNew York State Commissioner, Office of Addiction Services and Support (OASAS)Combating the Opioid Crisis in New York State: A Community Effort.

Daniel Rosa, MDBoard Certified Internist, Addiction Medicine Specialist, and Senior Medical Director, Acacia NetworkThe Neurobiology of Addiction.

James Hollywood, LCSWVice President of Residential Treatment, Samaritan Daytop VillageEvidence Based Treatment and Harm Reduction / What Social Workers Need to Know.

Q&A session and kosher dinner will follow keynote addresses

New York State Commissioner Arlene Gonzalez-Sanchez, MS, LMSW Office of Addiction Services and Support

Commissioner Arlene Gonzalez-Sanchez, MS, LMSW, was appointed New York State Commissioner of the Office of Addiction Services and Support (NYS OASAS) by Governor Andrew M. Cuomo on Jan. 3, 2011.

Commissioner Gonzalez-Sanchez has more than 30 years of experience in the fields of behavioral health services administration, health policy development, and medical research. Under the Commissioners leadership, OASAS works to realize her vision for transforming the current system of care for individuals with substance use disorders into a comprehensive, patient-centered and family-focused system. Commissioner Gonzalez-Sanchez serves on the Governors Medicaid Redesign Team (MRT) Behavioral Health Subcommittee. She is co-chair of the States Behavioral Health Services Advisory Council and a co-chair of the Responsible Play Partnership which addresses problem gambling in New York State.

On a national level, Commissioner Gonzalez-Sanchez serves as the First Vice President of the National Association of State Alcohol and Drug Abuse Directors (NASADAD) Board.

Commissioner Gonzalez-Sanchez holds a Master of Social Work degree in Administration and Community Organization from the Hunter College School of Social Work; and a Masters of Science degree in Cell Biology and a Bachelor of Science degree in Biology; both from Fordham University. She is a Licensed Master Social Worker.

Daniel Rosa, MDBoard Certified Internist, Addiction Medicine Specialist, and Senior Medical Director, Acacia Network

Dr. Daniel Rosa is an internal medicine specialist in the Bronx, New York where he was born and raised. He currently serves as the Senior Medical Director of Acacia Network (Clay Avenue Division), having previously been the Acting Chief Medical Officer. He also practices Emergency Medicine at Penn (Philadelphia VA Medical Center) on the weekends. He is a product of the public school system of NYC (Bronx) and furthered his education by graduating from Fordham University and then was accepted to the Harvard Medical School where after graduating he completed his residency in NYC. He has over 34 years of diverse experience in Internal Medicine, Addiction Medicine, HIV Medicine, and has been a member of the American College of Emergency Physicians (ACEP) for over 20 years. He also teaches the Advance Traumatic Life Support Course three times a year at the Westchester Medical Center and serves as facilitator for the review course for the California Society of Addiction Medicine (CSAM). Dr. Rosa also cooperates with other doctors and physicians in medical groups, including the Puerto Rican Organization To Motivate Enlighten And Serve Addicts Inc.

James Hollywood, LCSWVice President of Residential Treatment, Samaritan Daytop Village / Adjunct Professor Touro College

James Hollywood, LCSW, is Vice President of Residential Treatment and oversees the organizations 12 residential substance use disorder treatment programs, with over 950 beds and serving 3,000 clients each year. Over the course of his tenure in Samaritan Daytop Village, Mr. Hollywood led efforts to implement and sustain evidence based treatments to address addiction and co-occurring disorders that includes the use of psychometrically valid assessment tools and specific interventions to address the needs of those in residential treatment. Mr. Hollywood led the development and implementation of new residential clinical and administrative practices needed to create the new model of residential treatment, which include stabilization, rehabilitation and reintegration element of care. In addition to Residential Services, Mr. Hollywood manages two Criminal Justice programs: NYC DOC SMART Program, and Jails to Jobs. Mr. Hollywood brings more than 30 years of experience working in the fields of substance use, mental health and homeless services. Mr. Hollywood holds a BA in Sociology from St. John's University and graduated with an MSW from Hunter College School of Social Work.

Read the rest here:
A Community Day on the Opioid Addiction Crisis - Touro College News

The cells in our body are workshops that continuously operate to produce and process substances to keep us going. When a substance enters a cell for processing, it is surrounded by a portion of the cell's outer membrane to form a sac. The sac then buds off into the cell and becomes a vesicle containing the substance. This ingestion of substances by the cell is called endocytosis. The vesicle is then quickly merged with an endosome, an organelle also frequently referred to as a 'sorting station'. From the endosome, the substance is either recycled back to the cell membrane (for exiting the cell) or forwarded to a lysosome--a cell organelle containing enzymes for the breakdown of substances--for degradation. The substances entering a cell--and thereby an endosome--could be nutrients or signal molecules for processing, or even pathogenic viruses which can cause disease. It is therefore extremely important to fully understand the molecular basis of how endosomes are formed and maintained.

It is generally accepted by scholars in the field that these endosomes are formed and maintained by a mechanism in which vesicles traveling from the cell membrane constantly fuse into them. But in a recent study published in Communications Biology, a group of scientists from Japan and Austria, led by Prof Jiro Toshima from the Tokyo University of Science, claims that vesicles transported out of the Golgi--another crucial cell organelle--and not those from the cell membrane are more important for the formation and maintenance of endosomes. "We used our research to show that endocytic vesicle internalization is not essential, but that vesicle transport from the trans-Golgi network [TGN; the Golgi] is crucial," the team of scientists--comprising Makoto Nagano (Tokyo University of Science), Junko Y. Toshima (Tokyo University of Technology), Daria Elisabeth Siekhaus (Institute of Science and Technology, Austria) and Jiro Toshima--says.

They base their claim on the results of a series of experiments, involving the introduction of mutations or drugs (two drugs called Brefeldin A and Monensin) into yeast cells, to clarify the mechanism of endosome formation. As part of the experiments, they first used mutant yeast strains, which harbor mutations that cause defects in the endocytosis process; that is, they caused the ingestion of substances at the cell membrane to be hampered. Upon observation of the mutated cells, they found that the protein that mediates the formation of endosomes, Rab5, localized at the endosomes as per usual, leading to normal endosome formation.

They then introduced the two drugs into yeast cells to inhibit the transport of specific protein-containing vesicles from the Golgi. By doing so, they expected to find out whether these vesicles are required for the formation and maintenance of endosomes. They saw that smaller amounts of Rab5 localized on the endosomes, hampering endosome formation.

With further similar experiments, Prof Toshima and group saw that certain proteins, which are either resident in the Golgi or recruited to it, are transported from the Golgi to the endosomes where they activate Rab5 and spark the formation of endosomes. Deleting or deactivating the genes crucial to the transport of these proteins from the Golgi ultimately affects endosome formation.

Thus, considering all of these results together, it appears that endocytosis is not necessary for endosome formation and maintenance, but vesicle transport form the Golgi is.

Our results provide a different view of endosome formation and identify the TGN as a critical location for optimal maintenance and functioning of endosomes."

Prof Jiro Toshima, Tokyo University of Science

Given that endosomes are essential to the functioning of the cell and, by extension, the organism, understanding the mechanism of its upkeep is important. The results of this compelling study reveal but a fraction of this mechanism and much remains to be discovered. Even so, this advancement in the knowledge of one of the core pathways by which cells process substances in the body can lead to enhanced comprehension of the molecular basis of diseases that involve defective endosomes, thereby leading to better treatments for such diseases.

Source:

Journal reference:

Nagano, M., et al. (2019) Rab5-mediated endosome formation is regulated at the trans-Golgi network. Communications Biology. doi.org/10.1038/s42003-019-0670-5.

Link:
Study uncovers how the molecule-sorting station in our cells is formed and maintained - News-Medical.net

Heart failure (HF) is the most frequent cardiovascular diagnosis and exacts significant health and financial costs around the globe. It is estimated that at least 26 million people worldwide are living with HF, including nearly 6 million in the United States.1, 2 One in nine U.S. deaths in 2009 included heart failure as a contributing cause and about 50 percent of people in the U.S. with HF die within five years of diagnosis.2 The annual cost of HF-related healthcare services, medication and missed days of work is estimated at $40 billion in the United States and $108 billion globally.3, 4 Quality of life in HF patients is frequently worse than many other chronic diseases and comorbidities are common.5-7 The challenges of HF are expected to grow, as it is estimated that more than 8 million people in the United States alone will have HF by 2030.2 Current therapies improve quality of life in the short-term and have improved long-term survival but a significant number of patients have Class 3 HF despite optimal medical and device therapy. These patients have limited treatment options beyond heart transplant and left ventricular assist devices (LVAD). New therapeutic approaches that address the underlying causes of HF are needed to improve patient outcomes.

Heart failure is a complex disease process and multiple pathways contribute to its development and progression. Myocardial ischemia is frequently an issue in both ischemic and non-ischemic cardiomyopathy as well as HF with preserved and/or reduced ejection fraction. Myocardial ischemia results in insufficient oxygen and nutrients and leads to hypoxia, cardiomyocyte and fibrosis, which all contribute to the progression of heart failure. More effective angiogenesis may prevent this progression. Cell homing also plays a critical role, as injured cardiac tissue secretes factors that lead to the recruitment, proliferation, migration and differentiation of progenitor cells that can help repair tissue damage. Stromal cell-derived factor (SDF)-1 has been shown to play an important role in cardiac repair by mediating cell homing.10 Mitochondrial energy generation is also impaired in HF, leading to decreased contractility and adverse changes to cardiac architecture.11 Scar tissue formed in response to cardiomyocyte injury or death can compromise the hearts mechanical strength or electrical signaling results in myocardial infarction. Inflammatory responses to cardiac tissue damage can promote inappropriate and chronic inflammation and the expression of pro-inflammatory molecules that lead to pathologic changes to cardiac architecture.12, 13

These pathways offer a variety of potential new targets for therapeutic intervention to prevent the development and progression of HF. This opens the door to the development of novel therapies that address the underlying molecular and cellular causes of disease rather than treating HF symptoms alone.

After decades of development, gene-based therapies are now validated therapeutic modalities for the treatment of inherited retinal disorders and cancer and are undergoing clinical evaluation in a variety of inherited, acute and chronic diseases. Nearly two dozen single gene-based therapies for HF have been evaluated in clinical trials.14 Genes evaluated as monogenic gene therapy for HF in clinical trials include vascular endothelial growth factor (VEGF) and fibroblast growth factor type 4 (FGF4) to promote angiogenesis; adenylyl cyclase type 6 (AC6) and sarco/endoplasmic reticulum Ca2+-ATPase type 2 (SERCA2) to improve cardiac calcium homeostasis, which plays a critical role in the contraction and relaxation of heart muscle; and stromal cell-derived factor-1 (SDF-1) to improve cell homing and promote cardiac tissue repair. Late-stage trials of single gene therapies have yielded conflicting results, raising the question as to whether positively impacting a single pathway can be sufficient to overcome detrimental activity of other pathways that contribute to the development and progression of HF. Other potential limitations to HF therapies evaluated in clinical trials to date include the method of delivery, dose and the potency of vectors and gene products.

Given the multiple molecular and cellular pathways active in HF, a multi-gene approach to HF gene therapy may be needed. Simultaneously delivering multiple genes that target diverse HF-related pathways has the potential to improve cardiac biology and function. A triple gene therapy approach (INXN-4001, Triple-Gene LLC, a majority-owned subsidiary of Intrexon Corporation) is currently in clinical development, with each of the genes targeting a specific HF-related pathway. The investigational drug candidate INXN4001 vector expresses: the S100A1 gene product, which regulates calcium-controlled networks and modulates contractility, excitability, maintenance of cellular metabolism and survival; SDF-1a which recruits stem cells, inhibits apoptosis and supports new blood vessel formation; and VEGF-165 which initiates new vessel formation, endothelial cell migration/activation, stem cell recruitment and tissue regeneration. The hypothesis is that the simultaneous delivery of multiple genes in a single vector would more effectively improve multiple aspects of cardiac function compared with single gene therapy. It is delivered by retrograde coronary sinus infusion of a triple effector plasmid designed with a self-cleaving linker to constitutively express human S100A1, SDF-1a and VEGF 165. This route is designed to allow for delivery of a dose to the ventricle which may help achieve improved therapeutic effect.

Several preclinical studies have set the foundation on which to advance a triple gene therapy for HF into the clinic.15-17 Using in vitro studies, transfecting cells derived from patients with dilated cardiomyopathy with a triple gene combination demonstrated improvement in contraction rate and duration, to the levels demonstrated by the control cells and did not result in increased cell death compared to controls.15 Studies in an Adriamycin-induced cardiomyopathy rodent model demonstrated triple gene therapy increased fractional shortening and myocardial wall thickness compared to controls.16 In addition, retrograde coronary sinus infusion of INXN-4001 in a porcine model of ischemic HF resulted in a cardiac-specific biodistribution profile.17

A Phase 1 clinical study has been initiated to evaluate the safety of a single dose of triple gene therapy in stable patients implanted with a LVAD for mechanical support of end-stage HF. An independent Data and Safety Monitoring Board agreed to proceeding to the second cohort following review of the data from the first cohort in the multi-site study.18 The study is ongoing and final results will help to inform our understanding of the potential that multi-gene therapy may play in the treatment of HF.

The recent FDA approvals of gene therapies for an inherited retinal disease and cancer are evidence that gene therapy is a valid therapeutic strategy. Realizing the potential of gene therapy in HF will require appropriately designed clinical trials, but several interesting approaches currently in development may prove to be effective. The results of the initial investigational drug INXN-4001 Phase 1 trial should provide insight into the safety of combining S100A1, SDF-1a and VEGF-165. Evaluation of additional multi-gene combinations will also be important for understanding which targeted pathways yield the greatest effects with respect to relevant clinical endpoints. Continued refinement and optimization of vector design and delivery methods will also be important for advancing further HF gene therapies from bench to bedside.

Read more from the original source:
The Heart of the Matter: Leveraging Advances in Cardiac Biology to Innovate Gene-Based Therapies for Heart Failure - Physician's Weekly

Following is a transcript of the video.

Joe Avella: These gourmet dishes not only taste amazing but also give you a euphoric feeling because they are infused with cannabis. Chris Sayegh, aka The Herbal Chef, is a pioneer of cannabis-infused fine dining. Through his catered events, he's raised the bar on what is possible when mixing cannabis and high-end cuisine.

Chris Sayegh: It's more of a slight elevation rather than a overwhelming experience, and we have measurements in place from our staff to our food so that you don't get overwhelmed. I'm gonna show you how we incorporate CBD, THC, and terpenes throughout the meal.

Joe: Great.

Chris: Yeah.

Joe: His hard work and vision are paying off. He recently announced the opening of Herb in Los Angeles, a one-of-a-kind restaurant that will serve his cannabis-infused cuisine. We visited Chris at his home to learn more about his culinary cannabis journey.

Chris: I was studying molecular cell biology, going to medical school, but I wanted to be in preventative health. So, with that, I left, and I started to cook in kitchens.

Joe: Chris experimented on his own with cannabis in cooking, making elaborate, beautiful meals with minor THC dosage because he wanted to actually enjoy his meal instead of feeling too heavy or high.

Chris: The most paramount difference for all of this is what infusion is verses dosage. Infusion is putting a herb into a fat and then heating it up, and that infuses flavor and the THC and CBD into that. Great. Everybody and anybody can do that. What dosage is is infusing the herb and the flavor and the components into the fat at a consistent measurement. So, that way, you can know exactly how much THC, CBD, and terpenes you're providing to the customer in the meal.

Joe: Chris uses a nanotechnology that allows the THC and cannabinoids to absorb more efficiently into the body. Adding the cannabinoids doesn't affect the cooking process much, but a crucial step is at a stage where the lipids, or fats, will homogenize properly for even THC distribution.

Chris: If you don't homogenize correctly, which means to mix everything thoroughly and spread it out evenly, then one person's gonna get a big glob of the THC and another person's not gonna get anything. One person is gonna be in a fetal position and the other person is gonna be chilling, saying, "Why did I just pay for nothing?"

Joe: Chris was kind enough to whip up a few dishes for us to give us an overview of what he offers at his dining events. The events are usually 10 courses and include wine and spirits.

Chris: One of them is going to be a crab salad with a tabbouleh relish. And then there's going to be Wagyu dusted with caramelized onion and charcoal powder. And then there is a striped sea bass with a Broccolini and dashi soda. There is going to be a palate cleanser with peach and cream. For a vegetable course, we're going to do a mille-feuille of different root vegetables along with a compound black pepper and thyme butter. The whole idea is to use cannabis in all of its different forms, showcasing what we're able to do as we strive and push forward to hopefully earn a Michelin star in the future.

Joe: At a Herbal Chef event, each diner starts with a questionnaire to help Chris and his team gauge their tolerance and plan that diner's specific dosage.

Chris: So, we have many first-timers come to our events and to eat with us. Their biggest apprehension across the board is, "I've never tried it; I don't want to get too high." And it's totally reasonable. The whole idea is we want to take you along a journey. We want to take you along an experience rather than you go out for a quick bite to eat.

See the original post:
People pay $500 for these custom cannabis-infused meals - INSIDER

Upon meeting for the first time at a dinner at Stanford earlier this year, Fei-Fei Li and Jennifer Doudna couldnt help but note the remarkable parallels in their experiences as scientists.

Stanfords Fei-Fei Li and Jennifer Doudna of UC Berkeley will discuss the ethics of artificial intelligence and CRISPR technology. (Image credit: Getty Images)

Both women helped kickstart twin revolutions that are profoundly reshaping society in the 21st century Li in the field of artificial intelligence (AI) and Doudna in the life sciences. Both revolutions can be traced back to 2012, the year that computer scientists collectively recognized the power of Lis approach to training computer vision algorithms and that Doudna drew attention to a new gene-editing tool known as CRISPR-Cas9 (CRISPR for short). Both pioneering scientists are also driven by a growing urgency to raise awareness about the ethical dangers of the technologies they helped create.

It was just incredible to hear how similar our stories were. Not just the timing of our scientific discoveries, but also our sense of responsibility for the ethics of the science are just so similar, said Li, who is a professor of computer science at Stanfords School of Engineering and co-director of the Stanford Institute for Human-Centered Artificial Intelligence (HAI).

The ethical angle to what we were doing was not something that either of us anticipated but that we found ourselves quickly drawn to, said Doudna, who is a professor of chemistry and of molecular and cell biology at the University of California, Berkeley.

The echoes between Li and Doudnas lives were also not lost on the dinner host that night, Stanford political science professor Rob Reich, who invited the pair to resume their conversation in public. Their talk, titled CRISPR, AI, and the Ethics of Scientific Discovery, will take place at Stanford on Nov. 19 and will be moderated by Stanford bioengineering professor Russ Altman(livestream will be available here).

The event is organized by the Stanford McCoy Family Center for Ethics in Society and HAI and is part of the Ethics, Society & Technology Integrative Hub that arose from the universitys Long-Range Vision.

The subject of the lecture hits the sweet spot of what the Integrative Hubs work is about, which is to cultivate and support the large community of faculty and students who work at the intersection of ethics, society and technology, said Reich, who directs the Center for Ethics in Society and co-directs the Integrative Hub.

I cant think of two better people to engage in a conversation and to really take seriously these questions of how, as you discover the effects of what youve created, do you bring ethical implications and societal consequences into the discussion? said Margaret Levi, a professor of political science at Stanfords School of Humanities and Sciences. Levi is also the Sara Miller McCune Director of the Center for Advanced Study in the Behavioral Sciences and co-director of the Integrative Hub.

Fei-Fei Li is a professor of computer science and co-director of Stanfords Institute for Human-Centered Artificial Intelligence. (Image credit: L.A. Cicero)

In 2006, Li wondered if computers could be taught to see the same way that children do through early exposure to countless objects and scenes, from which they could deduce visual rules and relationships. Her idea ran counter to the approach taken by most AI researchers at the time, which was to create increasingly customized computer algorithms for identifying specific objects in images.

Lis insight culminated in the creation of ImageNet, a massive dataset consisting of millions of training images, and an international computer vision competition of the same name. In 2012, the winner of the ImageNet contest beat competitors by a wide margin by training a type of AI known as a deep neural network on Lis dataset.

Li immediately understood that an important milestone in her field had just been reached, and despite being on maternity leave at Stanford, flew to Florence, Italy, to attend the award ceremony in person. I bought a last-minute ticket, Li said. I was literally on the ground for about 18 hours before flying back.

Computer vision and image recognition are largely responsible for AIs rapid ascent in recent years. They enable self-driving cars to detect objects, Facebook to tag people in photos and shopping apps to identify real-world objects using a phones camera.

Within a year or so of when the ImageNet result was announced, there was an exponential growth of interest and investment into this technology from the private industry, Li said. We recognized that AI had gone through a phase shift, from being a niche scientific field to a potential transformative force of our industry.

The field of biology underwent its own phase shift in the summer of 2012 when Doudna and her colleagues published a groundbreaking paper in the journal Science that described how components of an ancient antiviral defense system in microbes could be programmed to cut and splice DNA in any living organism, including humans, with surgical precision. CRISPR made genomes as malleable as a piece of literary prose at the mercy of an editors red pen, Doudna would later write.

CRISPR could one day enable scientists to cure myriad genetic diseases, eradicate mosquito-borne illnesses, create pest-resistant plants and resurrect extinct species. But it also raises the specter of customizable designer babies and lasting changes to the human genetic code through so-called germline editing, or edits made to reproductive cells that are transmitted to future generations.

This bioethics nightmare scenario was realized last fall when a Chinese researcher declared that he had used CRISPR to edit the genomes of twin girls in order to make them resistant to HIV. Doudna decried the act but allows that her own views on germline editing are still evolving.

Ive gone from thinking never, ever to thinking that there could be circumstances that would warrant that kind of genome editing, she said. But it would have to be under circumstances where there was a clear medical need that was unmet by any other means and the technology would have to be safe.

Both Li and Doudna fervently believe in the potential of their technologies to benefit society. But they also fear CRISPR and AI could be abused to fuel discrimination and exacerbate social inequalities.

The details are different for CRISPR and AI, but I think those concerns really apply to both, Doudna said.

Rather than just leaving such concerns to others to work out, both scientists have stepped outside of the comfort of their labs and taken actions to help ensure their worst fears dont come to pass. I almost feel that at this point of history I need to do this, not that its my natural tendency, Li said. It really is about our collective future due to technology.

Both scientists have testified before Congress about the possibilities and perils of their technologies. Li also co-launched a nonprofit called AI4All to increase inclusion and diversity among computer engineers and she co-directs Stanford HAI, which aims to develop human-centered AI technologies and applications. Doudna spends significant time talking to colleagues, students and the public about CRISPR. In 2015 she organized the first conference to discuss the safety and ethics of CRISPR genome editing.

Because we were involved in the origins of CRISPR, I felt it was especially important for my colleagues and me to be part of that discussion and really help to lead it, Doudna said. I asked myself, If I dont do it, who will?

To read all stories about Stanford science, subscribe to the biweekly Stanford Science Digest.

Altman is the Kenneth Fong Professor of Bioengineering, Genetics, Medicine, Biomedical Data Science and host of the Stanford Engineering radio show The Future of Everything. Levi is a member of Stanford Bio-X, the Wu Tsai Neurosciences Institute, and the Stanford Woods Institute for the Environment. Li is the Sequoia Capital Professor at Stanford and a member of Stanford Bio-X and the Wu-Tsai Neurosciences Institute.

Read more here:
AI and gene-editing pioneers to discuss ethics - Stanford University News

Researchers strive to address societal health issues through gene editing

In October, three researchers at UC Davis were awarded a $1.5 million grant to fund their project which attempts to demonstrate the effectiveness of gene editing through use of CRISPR, a powerful technology that allows alteration of DNA sequences to change gene function.

This kind of design can help enhance personalized medicine, said R. Holland Cheng, a professor of molecular and cellular biology in the College of Biological Sciences. Specific patients with specific illnesses can be treated in specific ways.

Cheng, along with Kit Lam, a distinguished professor and chair of the Department of Biochemistry and Molecular Medicine in the School of Medicine, and David Segal, a professor in the Department of Biochemistry and Molecular Medicine, were awarded this highly competitive and sought-after grant from the National Institute of Health (NIH).

UC Davis is part of the NIHs Somatic Cell Genome Editing (SCGE) consortium which has awarded grants to 45 other research institutes across the nation so they can begin groundbreaking work on gene editing. Through this consortium, the NIH hopes to find an efficient and safe way to conduct gene editing. Research programs are investigating the best delivery mechanism as well as the most dynamic gene editing tool.

The major problem with gene editing currently is the inability of cells to be edited within a living organism. It has become fairly easy and efficient to edit genes in a cell culture outside of the body but extremely difficult to do the same processes inside the body. Cheng, Lam and Segal are focused on changing this.

The question is how to do it inside of an animal and eventually a human, Lam said.

They are answering this question by utilizing Chengs work in engineering a non-toxic nanoparticle that they hope can transport the gene editing tool CRISPR into the cells of a living organism. Cheng has been able to create a Hepatitis E viral nanoparticle (HEVNP) that when manipulated could be a delivery system for CRISPR. They plan to take this nanoparticle and encase CRISPR inside of it, producing a mechanism for delivery of CRISPR.

The Hepatitis E nanoparticle has the capacity to be a highly efficient way to deliver gene editing to cells in the body due to its unique nature. HEVNP is resistant to the gastric acid environment of the intestines and stomach, enabling it to survive once its entered the body. Given its resistant abilities, HEVNP can be taken orally, making it a useful form of medicine. If able to successfully get HEVNP to the target cells in the body and deploy CRISPR, gene editing abilities could drastically change.

The addition of a cell-type specific targeting ligand to the HEVNP would code the nanoparticle to deliver CRISPR to a specific cell. The abilities of this method to be precise and safe will determine its success.

With five years of funding from the NIH, these three researchers are eager to begin work on this project and see the strides that can be made in gene editing. They have impressive goals for this research, as it has the capacity to reshape medicine.

This will redefine precision medicine as currently there is broad medicine that can cause side effects to people and not be effective, yet by making it specialized it is becoming more precise and effective, Cheng said.

As more effective and safe tools to cure illnesses are being tested and created, the benefits to society could be expansive. With so much potential to help improve the health of society, the NIH is dedicated to coming to new solutions at a quick rate. All programs that received grants will be required to share and utilize the research occurring at other funded programs. The NIH is hoping to eliminate the private nature of research through enforcing the sharing of ideas, as scientists are often constrained by the institutions they work for. It is their hope that by having communication between the programs, positive results will arise faster.

I think this is great because scientists inherently want to work with each other but have real world concerns especially with money, Segal said.

The research results, when groundbreaking, can provide incredible monetary gains and credibility to the institutions that made the discovery. Ultimately, scientists collaborating with one another will serve society as people are able to benefit earlier from this innovative research.

We want the public to know that we are working in their best interest, Segal said.

The NIH grant is competitive and still the third research program to join the consortium at UC Davis. Innovation has never been more prevalent than in this field at UC Davis. With three different programs researching gene editing, UC Davis stands out as a hotspot for this field of research.

Written by: Alma Meckler-Pacheco science@theaggie.org

Read more:
UC Davis leads in innovative gene editing research with NIH grants - The Aggie

NOVEMBER 16 Once science fiction, the treatment of cancer using genetic modification is now a reality. Gene and immunotherapy are now bringing vast life-saving options for people diagnosed with cancer. But more needs to be done to raise awareness of this in Malaysia.

In Malaysia, more than 100,000 people have cancer and that is only registered figures. It is the fourth leading cause of death in the country. In 2018, the highest new cases in Malaysian females are due to breast cancer, and the highest for men are lung and colon cancer.

Cancer is the uncontrolled or unregulated growth of cells in our body. Gene therapy is the editing or replacement of genes in cells that have gone haywire. The easiest way to think about gene therapy is surgery. But instead of surgery on the whole person and being wheeled to the operating theatre, gene therapy is seen as surgery on specific problematic cells in our body.

While immunotherapy is a strategy in improving our immune system to recognise and attack cancer cells. It is similar to equipping our air force with the latest missiles and guiding system.

Gene therapy and immunotherapy make up a suite of advanced clinical approaches that are known as targeted or precision therapy. Advances in precision therapy in the next few years lends promise to do away with the need for chemotherapy and radiotherapy, in particular cancers detected in the early stages; as well as lends promise for where surgery is not possible.

Chemotherapy is often known as carpet bombing it takes out the enemy, but there is quite a bit of unwanted causalities. Thus, more traditional chemotherapies are known for side effects that include hair loss, mouth sores, nausea, bowel issues and skin problems.

Much of the advances in cancer treatment today are due to breakthroughs in genetics, immunology, virology, cell biology, and the related sciences. With the human genome mapped out, researchers have been successful in identifying specific genes that contribute to specific cancers.

Recently, our own Malaysian scientist Dr Serena Nik-Zainal was awarded the Dr Josef Steiner Cancer Research Prize Award 2019 for her breakthrough in holistic interpretation of the cancer genome. Based on her research, tumours can be analysed using new bioinformatics methods which will help with targeted therapies.

To simplify the complexities of gene therapy, there are three key aspects to this technique. We first need to know which gene is causing the type of cancer in question. We then need to know how to repair or replace this gene. And we also need to know what vehicle to transport this fix to the targeted cells.

One recent scientific advancement in gene therapy is known as CRISPR-cas9. CRISPR-cas9 is likened to a science-fiction scissors so small that it can cut and replace our genes that are defective. This tool is valuable for example in switching off a gene in cancer cells that continue to replicate and grow.

While one scientific advancement in immunotherapy is known as CAR T. Here, a persons T-cells are extracted, modified and placed back into the person so as it now has the ability to recognize and destroy cancer cells.

Scientists are still trying to address multiple challenges in the use of gene and immunotherapy. One such challenge in gene therapy includes finding an efficient vector or vehicle to deliver the gene fix to targeted cells. This is partly due to our immune system that can attack a foreign vehicle used in the transportation process.

Another challenge is in production, i.e. in taking what works in research labs and mass producing these therapies with high quality control. The latter contributes to the current high-cost of such advanced strategies to treat cancer.

No matter the challenges, advances in gene and immunotherapy continue exponentially around the world as confidence grows in breakthroughs achieved in treating cancer. In 2017 alone, 2,600 clinical trials were completed or were approved in 38 countries.

Examples of approved products available in the market include Gendicine (for head and neck cancer), Kymriah (for acute lymphoblastic leukaemia), Yescarta (for B-cell lymphoma), Olaparib (for breast and ovarian cancer), and Ibrance (for breast cancer).

This year a new drug called Larotrectinib has shown promise to treat a wide range of tumours. Known as tumour-agnostic, drugs such as Larotrectinib target common biochemical pathways across different types of tumours including sarcomas, brain, kidney, and thyroid tumours.

To better understand advances in cancer detection and treatment, the Department of American Canadian Education (ACE) at HELP University is organising a seminar-cum-forum on 16th November 2019 at their campus in Damansara.

Highly acclaimed speakers include Dr Lim Kue Peng of Cancer Research Malaysia and Dr Murallitharan of National Cancer Society Malaysia.

The event is open to all, including medical professionals, cancer survivors and the general public. For registration and further information, please go to https://helpxcancer.org or https://HELPUSCanada.weebly.com.

* Brendan J. Gomez has a first-class honours in genetics, and postgraduate degrees in psychology under the US Fulbright programme.

** This article is not meant as medical or pharmaceutical advice for cancer patients. Those seeking treatment need to consult with a medical professional, and preferably an oncologist.

*** This is the personal opinion of the writer or publication and does not necessarily represent the views of Malay Mail.

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Advanced life savers for cancer: From science fiction to reality Brendan J. Gomez - Malay Mail

Discover how RNA in situ hybridization assays are being used to advance research into treatments for Achilles tendon injuries

Orthopedic injury, specifically tendon injuries, are extremely prevalent. Depending on the severity of the injury, treatment ranges from rest to surgery. Often, tendon repair is a lengthy process that results in decreased mechanical function and can lead to chronic pain. The cell populations that make up the tendon, and the roles they play in the repair process, remain incompletely understood. Understanding the cellular makeup of tendons and its rolein repair could lead to improved treatments for tendon injuries.

In this exclusive SelectScience interview, we discuss this phenomenon with Jacob Swanson, a scientist in the lab of Dr. Christopher Mendiasin New York. Here, Swanson discusses the Mendias labs extensive research concerning the anatomy and physiology of the Achilles tendon, in both normal health and disease contexts. Swanson highlights how the use ofRNAscope technology has helped his work and why he hopes to continue to use this technology in future studies.

The Achilles tendon connects the calf muscle (the gastrocnemius and soleus) to the heel bone (the calcaneus). The role of the tendon is to transfer load between the soft tissue of the muscle and the hard tissue of the bone. The proximal side of the tendon attaches to the muscle and distal side attaches to the bone. Locating where different cell populations are in respect to this geometry is important when studying the tendon function.

JS:The main project that I've been working on is a descriptive study that aims to better understand the cells that make up the Achilles tendon. One major component of the tendon is a cell population called tenocytes, which are fibroblasts responsible for producing the collagen that makes up the tendon. However, the transcriptomes of cell types within Achilles tendon, including tenocytes, remain poorly characterized. To better understand these cell types, we used single-cell RNA sequencing technology (scRNAseq). Starting with Achilles tendon tissue, we enzymatically broke it down into a single-cell suspension and looked at the genetic information from each individual cell. We then clustered these cells using computational analyses to better understand the transcriptomic heterogeneity of cells in the Achilles. We then used RNAscope technology to histologically validate these cell populations

From this study, we identified interesting findings about fundamental tendon cell biology. We hope that this information can be used as an aid in future experiments and give us a better idea of how to assist the tendon-repair process.

JS:RNAscope technology allows one to visualize gene expression and RNA within cells in a broader histological context. For us, this means that we can see where the genes of interest are within the context of the Achilles tendon. We initially used immunofluorescent protein staining, but this proved ineffective because the markers often became incorporated into the extracellular matrix, making it hard to identify which specific cells these proteins came from. With RNAscope, we can directly probe for the mRNAs highlighted by RNAseq data within the cells, which allows us to more accurately identify and visualize where these novel cell populations are.

In our RNAscope protocol, we have several probes for genes based our scRNAseq data. First, we digest the cells slightly to allow the probe to enter the cells to hybridize to the RNA. Then there is a series of amplification steps that allow us to image three probes on a confocal microscope, cleave the fluorophore tag off, and then detect the next set of targets with the specified fluorophore tag. Theoretically, we can image up to 12 genes per sample all on the same section. This means we can get a profound amount of information on just one tissue section.

This makes RNAscope a powerful tool to visualize novel cell populations and the gene-specific probes allow us to see cell-to-cell heterogeneity. With this, you're able to see the difference in gene expression, even in cells that are right next to each other, which is difficult to detect with standard protein staining.

JS: In the past, it's been shown that the adaptive immune response is turned on in response to tendon injury, but the innate immune response hasnt been well studied before. In one study, we are focusing on this innate immune response, outlining possible mechanisms as to how the different cells are talking to each other within this response. We use healthy mice and perform Achilles tendon tear-and-repair surgeries. Over different time points, we monitor the various states of the immune response around the tendon, by collecting both tendon samples and samples from the lymph nodes that drain the tendons. We then analyze the different markers that are expressed at different times during the innate immune response using genetic and protein analysis, plus flow cytometry, to put together a picture of what happens following tendon injury.

JS: There is the potential to play with gene manipulation in future experiments. It could be that some of the cell types in Achilles tendons have positive effects in the injury process, but others might have negative effects. So, we could try to manipulate in both directions to redirect these cells during the injury course either by deleting or boosting the expression of a gene within the tendon. Additionally, our scRNAseq work thus far has been in mice, so it is important to also analyze the transcriptomes of human tendon cells to better understand how they may respond to injuries.

Explore more of the latest applications ofRNAscope technology:

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Musculoskeletal injury: New cellular targets to assist the tendon repair process - SelectScience

OXFORD, UK 13th November 2019 Arctoris Ltd, a fully automated drug discovery platform for virtual and traditional biotechnology companies, pharmaceutical corporations and academia, today announced the appointment of Professor John Mattick AO FAA FTSE FAHMS FRSN HonFRCPA GAICD as a Member of the Board of Directors, and Daniel Thomas PhD LCGI as Head of Discovery Biology. Arctoris is strengthening its senior team while the company is growing its commercial operations and service portfolio to enable more customers to rapidly reach their next drug discovery milestone.

Martin-Immanuel Bittner MD DPhil, Chief Executive Officer and Co-Founder of Arctoris, commented: I am delighted to welcome Professor Mattick and Daniel Thomas to our senior team. Their depth of expertise and experience will enable us to further develop our revolutionary drug discovery platform for customers and partners around the world. I look forward to working closely with them to enhance our novel capabilities and services.

As the former Chief Executive Officer of Genomics England and the former Executive Director of the Garvan Institute of Medical Research in Sydney, Professor Mattick is a leading expert on how the power of technological advances can transform the discovery and delivery of new therapeutic approaches. He has published over 300 research articles and reviews, which have been cited over 64,000 times, and has received numerous awards for his contributions to the field of molecular biology. With this significant scientific and strategic insight, his appointment to the board of Arctoris will support the development of the companys pre-optimised and fully validated research and development processes.

"I am delighted to join the Board of Directors of Arctoris. Its fully automated platform allows access to a wide range of methods and technologies, new levels of reproducibility, and exceptional data capture and integration capabilities that are unavailable elsewhere, which will transform biomedical research and drug discovery," said Professor John Mattick, Member of the Board of Directors, Arctoris.

Daniel Thomas, Head of Discovery Biology, Arctoris, added: Generating high-quality, reproducible data while balancing cost, scale and complexity is a fundamental challenge faced by the global drug discovery community today. Having experienced these pressures first-hand, I am committed to helping our customers and partners by configuring, running and monitoring experiments in real-time through Arctoris fully automated drug discovery platform.

Daniel Thomas joins Arctoris with more than 20 years experience working in early-stage small molecule drug discovery with GlaxoSmithKline. He brings with him an extensive theoretical and practical knowledge of assay development and mechanistic profiling together with an accomplished leadership track record developing trans-national matrix research teams. He also has significant experience in the implementation of transformative technologies and the delivery of key mechanistic data across a broad range of therapeutic areas.

For more information on how Arctoris is transforming drug discovery, please visit http://www.arctoris.com.

About Arctoris Ltd

Arctoris Ltd is an Oxford-based research company that is revolutionising drug discovery for virtual and traditional biotechnology companies, pharmaceutical corporations and academia. Arctoris has established the world's first fully automated drug discovery platform, offering pre-optimised and fully validated R&D processes for its partners and customers globally. Accessible remotely, the platform provides on-demand access to a wide range of biochemical, cell biology and molecular biology assays conducted by robotics, enabling rapid, informed decision-making in basic biology, target validation, toxicology and phenotypic screening. These assay capabilities are accessed using a powerful online portal that streamlines experiment planning, ordering, tracking and data analysis. Thanks to the Arctoris platform, customers can rapidly, accurately and cost-effectively perform their research and advance their drug discovery programmes.

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Arctoris Ltd Strengthens Senior Team to Continue the Development of its On-Demand Drug Discovery Services - BioSpace

Global Live Cell Imaging Marketwas valued at US$ 1.5Bn in 2017 and is expected to reach US$ XX Bn by 2026, at a CAGR of XX% during a forecast period.

Global live cell imaging market is majorly influenced by the growing incidence of chronic diseases and the consistent need for swift diagnostic techniques. Availability of exact and accurate live cell imaging techniques also help in accelerating drug discovery processes and other biotechnology research.

Growth in expenditure and funding for the development of advanced cell imaging is further expected to boost the live cell imaging market in the future. It is also observed that collaborations of market players with research and academic institutions to develop and introduce breakthrough products have recently gained pace. Small players are being increasingly acquired by large incumbents for procurement of breakthrough technologies to secure their stronghold in the market.

Fluorescence recovery after photobleaching is the most commonly used technique for live cell imaging. The technique has found rapid adoption in genetic targeting peptides and appropriately offers a determination of spatial proximity at a protein level that is not possible through fluorescence microscopy. Rapid introduction of FRET systems with an insight to offer better cell imaging techniques will so determine the major market trends.

Cell biology segment is leading the application owing to the increasing number of researchers working on molecular interaction networks. Innovations, for instance, filter techniques and advanced illumination devices further enable the procedure. Cell biologists use live cell imaging to understand the fundamental cellular structures and their interaction on the tissue level. Benefits are clarity of structural components and spatial heterogeneity of a cell offered by live cell imaging are expected to further boost the market.

North America dominated by market share in 2017 closely followed by Europe. Substantial investments and funding available for research in this field is the key driver in the North America region. The growing adoption of live cell imaging by research laboratories and academic institutions, particularly in the U.S. is one of the major factors driving market growth in this region.

One of the recent acquisition in the industry was done in March 2017 by Sartorius who agreed to buy Essen Bioscience in a transaction worth US$ 320Mn. Essen was energetic in developing equipment, reagents, and software.

Nikon Corporation Company has strategic partnerships with research groups to gain professional expertise. They have established imaging centers and offer microscopes, automation, software, and support to various institutes, for instance, Harvard Medical School.

The objective of the report is to present a comprehensive assessment of the market and contains thoughtful insights, facts, historical data, industry-validated market data and projections with a suitable set of assumptions and methodology. The report also helps in understanding Global Live Cell Imaging Market dynamics, structure by identifying and analyzing the market segments and project the global market size.

Further, the report also focuses on the competitive analysis of key players by product, price, financial position, product portfolio, growth strategies, and regional presence. The report also provides PEST analysis, PORTERs analysis, SWOT analysis to address the question of shareholders to prioritizing the efforts and investment in the near future to the emerging segment in the Global Live Cell Imaging Market.

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Scope of Global Live Cell Imaging Market

Global Live Cell Imaging Market, by Product & Service

Instruments Consumables Software ServicesGlobal Live Cell Imaging Market, by Application

Cell Biology Stem Cells Developmental Biology Drug DiscoverGlobal Live Cell Imaging Market, by End User

Pharmaceutical & Biotechnology Companies Academic & Research Institutes Contract Research OrganizationsGlobal Live Cell Imaging Market, by Region

North America Europe Asia Pacific Middle East and Africa South AmericaKey players operating in Global Live Cell Imaging Market

Danaher Corporation Carl Zeiss AG Nikon Corporation Olympus Corporation Perkinelmer GE Healthcare Bruker Thermo Fisher Scientific Sartorius AG Biotek Instruments Etaluma Cytosmart Technologies Nanoentek

MAJOR TOC OF THE REPORT

Chapter One: Live Cell Imaging Market Overview

Chapter Two: Manufacturers Profiles

Chapter Three: Global Live Cell Imaging Market Competition, by Players

Chapter Four: Global Live Cell Imaging Market Size by Regions

Chapter Five: North America Live Cell Imaging Revenue by Countries

Chapter Six: Europe Live Cell Imaging Revenue by Countries

Chapter Seven: Asia-Pacific Live Cell Imaging Revenue by Countries

Chapter Eight: South America Live Cell Imaging Revenue by Countries

Chapter Nine: Middle East and Africa Revenue Live Cell Imaging by Countries

Chapter Ten: Global Live Cell Imaging Market Segment by Type

Chapter Eleven: Global Live Cell Imaging Market Segment by Application

Chapter Twelve: Global Live Cell Imaging Market Size Forecast (2019-2026)

Browse Full Report with Facts and Figures of Live Cell Imaging Market Report at:https://www.maximizemarketresearch.com/market-report/global-live-cell-imaging-market/28816/

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Global Live Cell Imaging Market : Industry Analysis and Forecast (2018-2026) - The Market Expedition