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The Future of Medicine: CRISPR, Drug Prices & Gene Therapy

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  • The Future of Medicine: CRISPR, Drug Prices & Gene Therapy

    1:12

    01:55 Future of Medicine
    14:06 Future of healing
    27:14 Future of Diagnosis
    38:08 Future of Babies
    49:36 Future of Drugs

    What Happens Next examines the future as we confront massive technological transformations in central aspects of daily life. In this episode, we focus on water, food, work, driving, meat, and fact.

    What Happens Next is a production by RetroReport:

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  • Professor Jennifer Doudna - CRISPR-Cas9: Genome Editing and the Future of Medicine

    1:43

    Professor Jennifer Doudna is an American biochemist. She is Li Ka Shing Chancellor Chair Professor in the Department of Chemistry and the Department of Molecular and Cell Biology at the University of California, Berkeley. Doudna has been an investigator with the Howard Hughes Medical Institute since 1997, and since 2018 she has held the position of senior investigator at the Gladstone Institutes, as well as that of professor at the University of California, San Francisco.

    Professor Doudna has been a leading figure in what is referred to as theCRISPR revolution for her fundamental work and leadership in developing CRISPR-mediated genome editing. In 2012, Professor Doudna and Emmanuelle Charpentier were the first to propose that CRISPR-Cas9 could be used for programmable editing of genomes, which is now considered one of the most significant discoveries in the history of biology. They were awarded the 2020 Nobel Prize in Chemistry for their pioneering work.

    This is the CPM Dr Stanley Ho Memorial Lecture.

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  • Gene Therapy Basics

    3:30

    Gene therapy has been studied for more than 40 years and can help stop or slow the effects of disease on the most basic level of the human body—our genes. And to understand how it works, we’ll start at the basics.

  • CRISPR: Gene editing and beyond

    4:32

    The CRISPR-Cas9 system has revolutionised gene-editing, but cutting DNA isn’t all it can do. From turning gene expression on and off to fluorescently tagging particular sequences, this animation explores some of the exciting possibilities of CRISPR.

    Download a poster on ‘The expanding CRISPR toolbox’ here:

    Produced with support from Dharmacon:

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  • BREAKTHROUGH: Scientists Reverse Blindness CRISPR Technology

    14:24

    CRISPR Gene editing therapy is used for the first time in living humans with amazing results.

    0:00 Introduction
    0:53 What is CRISPR?
    2:05 How Does CRISPR work?
    3:18 The Experiment
    5:30 The Results
    9:31 Shortcomings
    10:34 The Future
    12:00 Caution
    13:02 Conclusions

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  • The Realities of Gene Editing with CRISPR I NOVA I PBS

    19:03

    CRISPR gene-editing technology is advancing quickly. What can it do now—and in the future?

    The revolutionary gene-editing tool known as CRISPR can alter, add, and remove genes from the human genome. The implications are immense: It could help eliminate illnesses like sickle cell disease and muscular dystrophy, and could even allow us to alter the genes of future generations of humans, leading to so-called designer babies. But will this ever really happen?

    Medical journalist and pediatrician Alok Patel investigates the current state of CRISPR—starting with a bull calf named Cosmo. Patel discovers how scientists edited Cosmo’s genome so he would produce more male offspring, and what that means for humans. In conversation with scientists, artists, and ethicists, Patel explores what kind of gene editing is actually possible right now—and what we should be thinking about when we consider manipulating human traits and, ultimately, the human experience.

    PRODUCTION CREDITS:

    Hosted by Dr. Alok Patel

    WRITTEN, PRODUCED AND DIRECTED BY
    Caitlin Saks

    EDITED BY
    Robert Kirwan

    ASSISTANT EDITOR
    Jay Colamaria

    CAMERA
    Arlo Pérez

    UC DAVIS FIELD DIRECTOR / CAMERA
    Zachary Fink

    ASSOCIATE PRODUCERS
    Ana Aceves
    Christina Monnen
    Arlo Pérez
    Jay Colamaria

    PRODUCTION ASSISTANT
    Lorena Lyon

    INTERNS
    Jessie Hendricks
    Samuel Lipsey

    AMERICAN SIGN LANGUAGE / ENGLISH INTERPRETER
    Adam Bartley

    ANIMATION
    Edgeworx Studios

    ADDITIONAL ANIMATION
    2K-12 Studios
    Mitch Butler

    MUSIC
    APM

    ONLINE EDITOR AND COLORIST
    David Bigelow

    AUDIO MIX
    Chris Anderson

    ARCHIVAL MATERIAL
    AP Archive
    Broad Institute of MIT and Harvard
    Getty Images
    He Jiankui Lab / CC BY
    Penn Medicine
    Pond 5
    Storyblocks

    SPECIAL THANKS
    Mike Baylis -- Assisted in the set up of a remote interview, at no cost.
    Isaac Plant -- Reviewed script and graphics for scientific accuracy, at no cost.
    Elizabeth Delgado -- Reviewed script and graphics for scientific accuracy, at no cost.
    Dr. Samira Kiani -- Reviewed script and graphics for scientific accuracy, at no cost.
    Dr. Kim Thornton -- Interviewed but did not include in the final film, at no cost.

    Participants:
    Françoise Baylis
    Teresa Blankmeyer Burke
    Karmella Haynes
    Xavier LaPlante
    David Liu
    Bret McNabb
    Liana Novoa
    Alok Patel
    Ariana Pelaez
    Brianna Sapienza
    Niaz Uddin
    Alison Van Eenennaam

    Image credit: (DNA helix)

    © WGBH Educational Foundation

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  • Therapeutic Human Gene Editing with Dr. David Liu

    1:3:34

    Over the past five to 10 years, development in the field of human gene editing has advanced at an unprecedented speed. We are now living in an age where therapeutic human gene editing is already a reality for some of us. Today we are joined by Dr. David Liu to discuss these exciting developments. Dr. Liu is a Richard Merkin Professor, Director of the Merkin Institute of Transformative Technologies and Healthcare, Vice-Chair of the Faculty at the Broad Institute of Harvard and MIT, and the Co-founder or Founder of nine biotech or therapeutics companies. He's published over 195 scientific papers and is the inventor of over 75 issued patents. Today he introduces us to the three main forms of modern genome editing, how they work, their benefits, and their limitations. He explains how the development of CRISPR had an enormous impact on the reach and scope of this field and describes some of the massive developments achieved by his labs in base editing and prime editing. Tune in today to hear how these different types of modalities might be best suited for different types of therapeutic interventions and Dr. Liu’s thoughts on whether or not one method will replace another. Find out if either of these technologies is more amenable to addressing polygenic diseases, the roles and complications of viral and non-viral delivery vectors, and how Dr. Liu believes these technologies will extend in the future. For all this and more, tune in today!

    Key Points From This Episode:

    - Dr. David Liu’s background and some of his impressive credentials.
    - An introduction to gene editing and how Dr. Liu became interested in it.
    - The groundbreaking development made by Peter Dervan and how this influenced the field.
    - Dr. Liu’s Unifactor 2000 project to develop a series of RNA molecules that could target any site in the genome and why it failed.
    - An introduction to the three main forms of modern genome editing.
    - What zinc fingers are and how they can be used to bind a DNA sequence of one's choosing.
    - What tail nucleases are and how tail proteins can bind a DNA sequence of one's choosing.
    - The breakthrough that came with the CRISPR system and how it initiated this modern gene-editing renaissance.
    - How the ease and accessibility of CRISPR has had an enormous impact on the reach and the scope of these developments.
    - What base editing and prime editing are and how they work together.
    - Some of the outcomes that can occur when you cut the DNA in a chromosome.
    - The two realizations that motivated Dr. Liu’s lab’s developments of base editing and prime editing.
    - Dr. Liu explains in detail how base editors and prime editors change DNA.
    - How Dr. Liu and his lab took the development of the CRISPR approach to develop these new techniques and how this was influenced by Homology Directed Repair.
    - What different types of modalities may be best suited for different types of therapeutic interventions.
    - Why one would pursue a CRISPR double-stranded break-based method versus doing base editing or prime editing.
    - How the companies Prime Medicine and Beam that have licensed these methods work together in the best interests of the patient.
    - Dr. Liu’s thoughts on whether or not prime editing is going to replace base editing.
    - Insight into the need for PAM sequences in Cas9 and the advantage of prime editing as it does not need a specific PAM sequence.
    - How Dr. Liu believes these technologies will extend in the future.
    - Thoughts on whether or not one flavor of these technologies is more amenable to addressing polygenic diseases.
    - What a delivery vehicle is, the differences between viral and non-viral vehicles, and the complications of using viral delivery vectors.
    - Concerns with regard to immunogenicity and immune reaction from an LMP and how this relates to the COVID vaccine.

    Tweetables:

    “There are already humans walking this earth that have had their genomes modified, specifically in a way to counteract a genetic disease. That's really a pretty momentous era in the history of man.” — @davidrliu [0:10:37]

    “The motivation behind base editing was the simple recognition that for most genetic diseases, to study the disease or to treat the disease, we can't really do that well if we just mess up the gene. We need to actually precisely fix it.” — @davidrliu [0:13:10]

    “I think that's one of the potential downsides of viral delivery is that whenever you're introducing new DNA into a cell, you have to be very careful to minimize the possibility that the DNA gets in and initiates cancer.” — @davidrliu [0:56:33]

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  • Radical new gene therapy restores sight to patients with rare eye condition - BBC News

    3:19

    Scientists have been using a new form of gene therapy to treat a rare inherited eye condition which eventually causes severe tunnel vision.

    Scientists have been using the new treatment on patients to try to halt further loss of sight. And they've been astonished to find that it has actually improved their vision.

    Sophie Raworth presents BBC News at Ten reporting by medical editor Fergus Walsh.

    Please subscribe HERE

    #BBCNews

  • Gene Therapy -- The time is now: Nick Leschly at TEDxBoston

    15:27

    Forget the price. What's the value of that treatment?

    What if we could fix a patient's own cells and cure their disease once and for all instead of treating a chronic illness over their lifetime? The solution exists, but it's expensive. Nick Leschly, chief bluebird, explains how biotechnology can deliver corrective genes into a patient's diseased cells and disrupt the medical and insurance industries along the way

  • x
  • Genetic Engineering Will Change Everything Forever – CRISPR

    16:04

    Designer babies, the end of diseases, genetically modified humans that never age. Outrageous things that used to be science fiction are suddenly becoming reality. The only thing we know for sure is that things will change irreversibly.


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  • How Gene Editing Will Change The World

    8:06

    A revolution is taking place in science, one that could potentially cure all disease, nourish the starving millions, and ultimately transform humanity into a race of hyper-intelligent super-beings.

    Brand new techniques enabling scientists and clinicians to manipulate and change the very genetic code of life itself are coming on-stream fast – and the implications are awe-inspiring, if not a little frightening. So join us today as we peer down the microscope for a glimpse of just how gene editing can change the world.

    Back in 2018, a Chinese scientist called He Jianjui made headlines around the world by announcing the birth of the first ever gene-edited babies. An associate professor and biophysics researcher, He claimed to have used the sophisticated new DNA editing technique known as CRISPR to manipulate a gene called CCR5 in a pair of twin girls, named Nana and Lulu. This CCR5 gene codes for a particular protein that allows HIV to infect human cells. By disabling this specific gene, He claimed to have genetically engineered Nana and Lulu to be resistant to diseases.

    How Gene Editing Will Change The World

  • 2015 WMIF | Gene Editing, Gene Therapy, and the Eye as a Gateway

    50:08

    Jennifer Joe, MD, CEO, Medstro, Editor-In-Chief, MedTech Boston
    Katrine Bosley, CEO, Editas Medicine
    Eric Pierce, MD, PhD, Director, Retinal Degenerations Service, Massachusetts Eye and Ear; Solman and Libe Friedman Associate Professor of Ophthalmology, Harvard Medical School
    Philip Gregory, D. Phil, Senior Vice President, Research and CSO, Sangamo BioSciences
    David Meeker, MD, CEO, Genzyme

    Panel members detail why the future of CNS therapeutics is moving away from miracle drugs and gravitating towards personalized treatments using gene therapy. They also describe how the DNA-editing tool, CRISPR, is empowering scientists to make quick and inexpensive edits to a stretch of DNA with a level of ease and efficiency not previously possible.

  • Seeking to Enable a Future of Cell and Gene Therapies

    3:28

    Usman (Oz) Azam, MD, Global Head of Novartis’s Cell & Gene Therapies Unit, addresses the potential transformation in the practice of medicine in the form of cell and gene therapies. Dr. Azam explains the more integrated development process required for cell and gene therapies compared with the typically sequential drug development process and highlights Novartis’s current focus in the cell and gene therapy field.

    © 2015 Novartis AG

  • CRISPR gene-editing tool

    11:39

    Researchers are experimenting with CRISPR, a powerful tool that can alter the DNA of humans. The implications have scientists excited, but nervous.
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  • IGI Seminar Series – Correction of the Sickle Cell Disease Mutation with CRISPR-Cas9

    58:27

    UCLA project scientist Mark DeWitt discusses a combined IGI, UCLA, and UCSF effort in developing a CRISPR-based clinical product to correct sickle cell disease. Listen to Mark as he describes how his team went from a bench genome-editing protocol to an accepted Investigational New Drug (IND).

    IGI Seminar Series - May 4, 2021

  • Bill Gates: How Gene Editing, AI Can Benefit Worlds Poorest

    1:12:13

    This plenary lecture was taped in Seattle at the AAAS Annual Meeting on Feb. 14th, 2020. Bill Gates is co-chair of the Bill & Melinda Gates Foundation. In 1975, Bill Gates founded Microsoft with Paul Allen and led the company to become the worldwide leader in business and personal software and services. In 2008, Bill transitioned to focus full-time on his foundation’s work to expand opportunity to the world’s most disadvantaged people. Along with co-chair Melinda Gates, he leads the foundation’s development of strategies and sets the overall direction of the organization. In 2010, Bill, Melinda, and Warren Buffett founded the Giving Pledge, an effort to encourage the wealthiest families and individuals to publicly commit more than half of their wealth to philanthropic causes and charitable organizations during their lifetime or in their will. In 2015, Bill created the Breakthrough Energy Coalition, a group of individuals and entities committed to clean energy innovation, followed by Breakthrough Energy Ventures in 2016, an investor-led fund focused on providing patient capital to support cutting-edge clean energy companies.

  • Talks@12: The Promises and Perils of Gene Editing

    1:00

    Genome reading and editing are exponentially advancing technologies, putting tools such as next-generation sequencing and CRISPR at the forefront of medical research and practice. George Church, a leading innovator in this emerging field, will explore the many promising applications that could significantly impact human health and also discuss the ethical implications facing genetic engineering now and in the future.

    George Church, PhD
    Professor of Genetics
    Harvard-MIT Program in Health Sciences and Technology
    Harvard Medical School
    Director, Personal Genome Project

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  • From Sick Care to Preventative Care: Rethinking Medicine

    31:30

    Global healthcare systems are buckling under the strain of rising costs, aging populations and chronic disease. It’s far better to prevent disease than to treat people after they get sick. How can medicine move toward a new model of care? What is the role of revolutionary new technologies like mRNA? David Leonhardt, Senior Writer, The New York Times moderates a panel with Rachel Haurwitz, President and CEO, Caribou Biosciences, Bill Anderson, CEO, Roche Pharmaceuticals and Francis deSouza, CEO, Illumina.

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  • The Convergence of CRISPR and Human Stem Cells

    1:20

    Gladstone President Deepak Srivastava moderated a conversation with Jennifer Doudna and Shinya Yamanaka to discuss the potential impact of CRISPR and iPS cells over the coming decade and hear from the two world-class scientists about how they plan to shape the enormous opportunities that lie ahead.

    Yamanaka was awarded the Nobel Prize in Physiology or Medicine in 2012 for his discovery of induced pluripotent stem cells (iPS cells). And last year, Doudna received the Nobel Prize in Chemistry for her co-discovery of the CRISPR-Cas9 gene-editing technology. Arguably the two most impactful advances of this century, the discoveries by these Gladstone investigators are now accelerating our understanding and treatment of human disease at an unprecedented pace.

  • CRISPR Office Hours with Jennifer Doudna, Ph.D.

    51:27

    We’re excited to welcome Jennifer Doudna, Ph.D. to the final CRISPR Office Hours episode.

    Jennifer is a Professor and HHMI Investigator at UC Berkeley and Howard Hughes Medical Institute, and President and Chair of the Innovative Genomic Institute (IGI). Co-inventor of the CRISPR-Cas genome engineering technology, and the founder of numerous gene editing companies, her lab discovers and develops CRISPR systems and other RNA-guided mechanisms of gene regulation.

    Jennifer will discuss her involvement in developing novel CRISPR-based COVID-19 diagnostics, how she spearheaded the transformation of the IGI into a high-throughput COVID-19 testing facility, and how her lab continues to develop and characterize novel CRISPR nucleases.

    #CRISPROfficeHours

  • x
  • CRISPR Screening - The What, Why and How

    53:24

    Speaker: Benedict C. S. Cross, PhD, Team leader (Discovery Screening), Horizon Discovery

    CRISPR–Cas9 mediated genome editing provides a highly efficient way to probe gene function. Using this technology, thousands of genes can be knocked out and their function assessed in a single experiment. We have conducted over 150 of these complex and powerful screens and will use our experience to guide you through the process of screen design, performance and analysis.

    We'll be discussing:
    • How to use CRISPR screening for target ID and validation, understanding drug MOA and patient stratification
    • The screen design, quality control and how to evaluate success of your screening program
    • Horizon’s latest developments to the platform
    • Horizon’s novel approaches to target validation screening

    For more information see:

  • The Science Behind Gene Therapy

    56:05

    After decades of research and development, gene therapy is becoming a clinical reality in the US. With the first gene therapies already approved and many more in the pipeline, healthcare providers may soon gain the ability to treat more rare conditions.

    To address this exciting topic, NORD is hosting a 5-part series on gene therapy. In part-2 of the series, The Science Behind Gene Therapy, we will explore how gene therapies are developed, the ways in which evidence-based science guides manufacturers of gene therapies, how vectors deliver gene therapies to the cells and what happens inside the body after gene therapy is delivered.We will cover these topics and more in this free webinar on September 25.

    This webinar is perfect for patients, caregivers, advocates, students and the public.

    Webinar Speakers:
    Sven Kili, MD, Principal at Sven Kili Consulting Ltd., provides strategic consulting services to regenerative medicine companies.

    Tiffany Lucas, PhD is an FDA Product Reviewer in the Division of Gene Therapy in the Center for Biologics Evaluation and Research (CBER).
    Katie Kowalski, MPH is the Senior Manager for NORD’s Educational Initiatives.

  • Crispr-Cas9 explained: the biggest revolution in gene editing

    4:22

    Professor Jennifer Doudna, one the pioneers of Crispr-Cas9 gene editing explains how this important discovery enables precise changes to our DNA , which can be used to correct mutations that cause genetic disease and completely eradicate it from a germline. Doudna raises the 4 key issues of debate around this revolutionary discovery and suggests what will have the most immediate impact.
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  • Meet the biohacker using CRISPR to teach everyone gene editing

    6:07

    Meet the biohacker who wants to teach everyone how to edit genes

    Josiah Zayner is a biohacker who thinks everyone should be able to change their DNA with biotechnology called CRISPR. That’s why he founded a company called The ODIN, which sells do-it-yourself biotech kits that teach people how to genetically modify bacteria and frogs. It's DIY gene therapy.

    His company has sold tens of thousands of experiments using CRISPR, an inexpensive and precise gene-editing technology that has revolutionized the field.

    Find more on CRISPR at Quartz

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  • Feng Zhang: The Future of Gene Editing - Schrödinger at 75: The Future of Biology

    23:56

    Feng Zhang , is a McGovern Investigator and an Associate Professor in the Departments of Brain and Cognitive Sciences and of Biological Engineering. He is also a core member of the Broad Institute of MIT and Harvard. He joined MIT and the Broad Institute in 2011 and was awarded tenure in 2016. Feng Zhang grew up in Iowa after moving there with his parents from China at age 11. He received his A.B. in chemistry and physics from Harvard College and his PhD in chemistry from Stanford University. Zhang has received many awards for his work in genome editing and optogenetics, including the Perl/UNC Prize in Neuroscience (2012, shared with Karl Deisseroth and Ed Boyden), the NIH Director’s Pioneer Award (2012), the National Science Foundation’s Alan T. Waterman Award (2014), the Jacob Heskel Gabbay Award in Biotechnology and Medicine (2014, shared with Jennifer Doudna and Emmanuelle Charpentier), the Society for Neuroscience Young Investigator Award (2014), the Okazaki award, the Canada Gairdner International Award (shared with Doudna and Charpentier along with Philippe Horvath and Rodolphe Barrangou) and the 2016 Tang Prize (shared with Doudna and Charpentier). Zhang is a founder of Editas Medicine, a genome editing company founded by world leaders in the fields of genome editing, protein engineering, and molecular and structural biology.

  • Genomics, Big Data, and Medicine Seminar Series – George Church

    1:29:04

    George Church, Professor, Genetics at Harvard Medical School and Director, PersonalGenomes.org, speaks on Why Big Data Is Easier than Little Data.

    Introductions by Eric Schadt, Director, Institute for Genomics and Multiscale Biology and Jason Bobe, Director, Sharing Lab, Icahn Institute for Genomics and Multiscale Biology.

  • A conversation with Bill Gates and Francis Collins on global health and genomics at #ASHG17

    1:23

    Bill Gates, Co-chair and Trustee of the Bill & Melinda Gates Foundation, and Francis Collins, Director of the National Institutes of Health (NIH), hold a live symposium at ASHG - American Society of Human Genetics to address global health and genomics.

  • Gene Therapy: Your Questions Answered

    5:57

    NORD’s RareEDU™ released this video, Gene Therapy: Your Questions Answered, in order to address a vital topic to today's rare disease community. The goal of this video is to address the questions, hopes and concerns that patients and caregivers, across many different diseases, have about gene therapy. Since more than 80% of rare diseases are believed to be genetic, this video will serve as a helpful resource for the rare disease community.

  • CRISPR 2021 - Future Of Gene Editing and Gene Therapy

    8:56

    CRISPR 2021 future of gene editing and Gene Therapy is a video on the latest technology in gene engineering. CRISPR cas9 is a protein used in the technique. CRISPR cures include cancer, genetic disorders etc. CRISPR huntington's disease and CRISPR sickle cell anemia are well known cures, CRISPR blindness cure is also well known. This is also a video on CRISPR explained. CRISPR gene therapy could also reduce cost and help patients.

    Thanks for watching the video guys! I started beyond wonder to answer some questions that were always at the back of my mind. I thought organizing my thoughts and sharing them could help me and in the process help people who have the same or similar questions. I discuss some contemporary social issues in this channel. If you found value in this video please consider subscribing.

    Background tracks -

    1. Stuck In The Air - The Tower of Light

    2. Nameless - Gabrial Douglas

    3. Haunted Forest - TrackTribe

    4. Atlantis by Audionautix

    Atlantis by Audionautix is licensed under a Creative Commons Attribution 4.0 license.

    Artist:

    Regarding the use of resources in the video -

    Most, if not all the resources used in this video are either licensed as creative commons or require attribution. I may have also used some copyrighted content, in that case, please note that these snippets are of minimal nature and are not intended to harm the original owners. And, the usage of these snippets comply with the fair dealing exception provided under sec. 52 of the copyright act. If you think I've wrongfully used any video, do mention the same in comments or contact me through Gmail - beyondwonder.yt@gmail.com. I've linked the sites that contain these resources below.

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  • The future of medicine

    8:04

    ABC News’ Linsey Davis examines the future of medicine, from how we visit the doctor to what’s ahead on vaccine development, and speaks with the University of Pennsylvania’s Dr. Ezekiel Emanuel.

    ABC News Live Prime, Weekdays at 7EST & 9EST

    WATCH the ABC News Live Stream Here:
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    #ABCNLPrime #Medicine #Future #VaccinationDevelopment #UniversityOfPennsylvania

  • Intellia’s Modular CRISPR/Cas9 Gene Editing Platform for the Treatment of Disease

    1:4:42

    At Intellia, we are building a full-spectrum, product-driven biotechnology company focused on our mission of developing and commercializing potentially curative genome editing treatments that can positively transform the lives of people living with severe and life-threatening diseases. On the in vivo side, our systemic lipid nanoparticle or LNP-based delivery system has the potential to unlock treatment of genetic diseases by selectively knocking out disease-causing genes, introducing targeted insertion of a functional gene or both. We are also focused on engineering T cell therapies to provide them with particular enhanced attributes that may enable them to more effectively treat oncological and immunological diseases. Our approach is designed to improve safety and efficacy by engineering cell therapies that are more precise, potent and persistent. The OTS webinar will focus on Intellia’s in vivo platform and pipeline, highlighting the paths to gRNA selection, preclinical pharmacology and the clinic.

    Oligonucleotide Therapeutics Society


    Intellia Therapeutics

  • Gene Editing

    2:14

    The ability to precisely edit the genome of a living cell holds enormous potential for accelerating life science research and biotechnology and even treating human disease. At the Broad Institute, researchers are pioneering the development and sharing of tools, such as CRISPR-based editing and genome engineering systems. These tools enable scientists worldwide to probe the genetic underpinnings of biological systems; quickly create cell and animal models for research; design low-cost diagnostics; precisely and simply edit the DNA of living things, from plants to people; and perhaps one day develop a powerful therapeutic approach.

    Copyright Broad Institute, 2019. All rights reserved.

  • Why CRISPR-Cas9 Is So Powerful: Gene Editing

    12:14

    Let's talk about gene editing (or genome engineering, however you prefer to call it). What a good time to be alive.

    We're witnessing how biotechnology has advanced to a point where editing our genome is not only possible but also practical, fast, cheap, and relatively easy. And all thanks to the biggest game-changer: CRISPR-Cas9. In this video, we'll talk about what gene editing is and the role the relatively recently discovered system CRISPR-Cas9 plays.

    00:00 Introduction
    01:09 Possible applications of CRISPR in health
    01:47 CRISPR’s role in gene editing
    03:02 Where does CRISPR come from?
    04:26 How can it be used to edit genes?
    04:43 Some scientific gossip
    06:13 What makes CRISPR so powerful?
    07:23 Repair mechanisms
    09:05 Is it all good news?


    Some of the sources and interesting links:












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    See you next time :)

  • The Future of Medicine

    55:15

    Former CEO and Chairman of Varian Medical Systems Richard “Dick” Levy, Managing Director of Sanderling Ventures Tim Mills, and School of Medicine Professor Mildred Cho are interviewed by instructor Ernestine Fu. In the last 50 years, we have seen incredible innovations in medicine. We have been able to eradicate smallpox, learn how to transplant organs, and even create artificial hearts. While these are all impressive, we still have a long way to go. The future of medicine will have even more profound impacts on our daily lives. The speakers explore recent advances in medicine, inefficiencies that must be addressed, and what the future holds for us.

    Course Description: The next wave of technological innovation and globalization will affect our countries, our societies, and ourselves. In the next economy, we will see a fusion of technologies and research that is blurring the lines between the physical, digital, and biological spheres.

    We will see extraordinary technology advances that are commensurate with those of the first, second, and third industrial revolutions. These earlier industrial revolutions introduced the use of steam power to mechanize production, development of groundbreaking inventions in transportation, telecommunications, and manufacturing, as well as more recently, the introduction of the digital era.

    As the next revolution is transforming humanity, MED/CEE 214: Frontier Technology explores the development and application of key emerging technologies in society: AI/ML; Smart Cities and Urban Mobility; Advanced Life Sciences; Telecommunications with 5G; and Data Privacy, Ethics, and Policy.

  • The First Retinal Gene Therapy Trial

    3:03

    Among the many hoped-for benefits of the revolutionary gene-editing technology CRISPR is treating, or even curing, diseases. At the 2016 World Science Festival, ophthalmologist and researcher Stephen Tsang discussed how CRISPR is already improving sight in patients with retinal degeneration.

    Excerpt from – Salon: Game Change - CRISPR's Brave New World
    Watch the full program here:

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  • CRISPR Breakthroughs for Neurological Disease

    58:07

    In this roundtable discussion, Jennifer Doudna, Stephen Hauser, and Ehud Isacoff discuss the promise and challenges of creating new therapies for neurological diseases and bringing them to the clinic, as well as the need to develop cures that are accessible and affordable.

  • Future of Medicine | Daniel Kraft | SingularityU Japan Summit

    48:03

    SingularityU Japan Summit 2017

  • Making the Cut | Session 1: Science and Society || Radcliffe Institute

    1:38:14

    The 2019 Radcliffe Institute science symposium is on gene editing, a technology that enables scientists to change an organism’s DNA. Leading international scientists, clinicians, and ethicists gather to explore case studies of particular gene therapies and consider the legal and bioethical implications of this research.

    WELCOME
    Tomiko Brown-Nagin, dean, Radcliffe Institute for Advanced Study; Daniel P.S. Paul Professor of Constitutional Law, Harvard Law School; and professor of history, Harvard Faculty of Arts and Sciences

    FRAMING REMARKS (04:41)
    Immaculata De Vivo, life sciences advisor, Radcliffe Institute for Advanced Study; professor of medicine, Harvard Medical School; and professor of epidemiology, Harvard T.H. Chan School of Public Health

    SESSION 1: SCIENCE AND SOCIETY (11:47)

    Introduction by moderator: Charmaine DM Royal, associate professor of African & African American studies, biology, global health, and family medicine & community health, Duke University

    SCIENCE KEYTNOTE (17:22)
    Sylvain Moineau, Canada Research Chair in Bacteriophages, Department of Biochemistry, Microbiology, and Bio-informatics, Faculty of Science and Engineering, Université Laval (Canada)

    SOCIETY KEYNOTE (37:38)
    Jonathan Kimmelman, James McGill Professor and director of the Biomedical Ethics Unit, Department of Social Studies of Medicine, McGill University (Canada)

    PANEL DISCUSSION (53:32)
    AUDIENCE Q&A (1:14:33)

    For information about the Radcliffe Institute and its many public programs, visit

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  • Applications of Human Genetics in Drug Discovery to Present and Future Indications

    52:09

    Presented by Lon Cardon, Ph.D. (GlaxoSmithKline) at the PGRN Symposium on October 17, 2016 in Vancouver, BC.

    JOIN the PGRN:


    ​All individuals interested in pharmacogenomics research may apply.

  • The Future of Genome Editing with Professor David Liu

    55:54

    Today’s guest on the For Your Innovation Podcast is Professor David Liu, who will be sharing his expertise on the subject of genome editing, the ability to make highly specific changes in the DNA sequence of a living organism, essentially customizing its genetic makeup. David is a Richard Merkin Professor, Director of the Merkin Institute of Transformative Technologies in Healthcare, and Vice-Chair of the Faculty at the Broad Institute of Harvard and MIT, as well as the scientific founder or cofounder of six biotechnology and therapeutics companies, including Editas, Exo Therapeutics, Beam Therapeutics, and Prime Medicine. He himself went to Harvard and graduated first in his class, and he has since published more than 195 scientific papers and is the inventor of 75+ issued U.S. patents.

    Professor Liu’s research integrates chemistry and evolution to illuminate biology and enable next-generation therapeutics and, in this episode, he shares some of his insights into base editing and prime editing and their advantages when compared with CRISPR, specifically the advantages of precisely fixing rather than disrupting genes, which could be a life-saving solution for a huge number of genetic conditions. He also touches on the next frontier of proteome editing and explains the role that Botox plays, his surprising involvement in developing a system to beat Blackjack, and his predictions for the future of gene editing therapeutics, some of which might sound like science fiction! Tune in today to learn more.

    Key Points From This Episode:

    • David explains what base editing is: a strategy for changing the genome instead of cutting the DNA double helix.
    • Some advantages of base editing, including precisely fixing rather than disrupting a gene.
    • How prime editing factors into this, enabling all 12 possible base-to-base changes as opposed to the four of base editing.
    • Hear about the background of these discoveries and David’s involvement in developing them.
    • The relationship between Beam and Prime Medicine; how they leverage technologies and experience to bring prime editing to patients.
    • David touches on some of the potential applications of genome editing in agriculture.
    • David’s thoughts on how quickly costs for these therapeutics will decline, based on their stunning rate of progress.
    • Whether gene editing therapeutics could dominate or replace small molecule therapeutics.
    • DNA templated synthesis and DNA encoded chemistry and their synergy with gene editing.
    • How he divides his time and responsibilities between his lab and his various companies.
    • A fun fact about David: he taught a class on developing systems that could beat Blackjack!
    • Finally, hear David’s predictions for gene editing therapeutics over the next five to 10 years.

    Tweetables:

    “For most human genetic diseases, you need to precisely fix a gene in order to have the anticipated therapeutic benefit, as opposed to disrupting the gene.” — @davidrliu

    “I care deeply about making our science transition from the pages of a journal to some societal benefit, whether it's more nutritious crops or new medicines for diseases that can't be treated otherwise.” — @davidrliu

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  • CRISPR, Sickle Cell Disease, & Society: A VR Explainer & Ethics Discussion

    1:11:45

    This is a recording of an event held on October 23rd, 2020. The program begins at the 7:25 mark.

    Learn about DNA editing technology! Engage with an expert panel on the ethics of treating a disease that affects marginalized communities.

  • 2021 Reuters Cell & Gene Therapy

    13:20

    Global President of Pfizer Rare Disease, Suneet Varma, delivers a keynote ‘What the COVID-19 vaccines can teach us about gene therapies for rare diseases’ at the 2021 Reuters Cell & Gene Therapy USA.

    NOTE: Video was originally presented on September 28, 2021.

  • Friends Lunch with a Member - Eben Kirksey

    35:03

  • The Future of PKU Drug Development

    39:05

    Betsy Bogard describes how patient organizations can play a
    vital and central role in the drug development process. She will review the process, findings, and outcomes of this project for the future of PKU.

  • Advancing Clinical Research of Gene Therapies in Brazil

    4:21:08

    This event is in partnership between ASGCT and the Brazilian Association of Cellular and Gene Therapy. The goal of this program is for speakers with experience in clinical development to share their experiences and insights on factors to consider to successfully initiate clinical trials of in vivo and ex vivo gene therapies in Brazil. Stimulating such additional clinical research is an important step in expanding availability and access to these therapies for patients in need in the area.

  • Medical breakthroughs to watch for in 2016

    3:24

    The top health stories in 2015 included rising drug prices, the first genetically-modified salmon, the first sex drug for women and processed meat declared a cause of cancer. Now, we’re looking at the most promising medical advances in the new year. From gene editing technology CRISPR to elephants helping to fight cancer, Dr. David Agus joins “CBS This Morning” to make predictions for 2016.

  • CAGS Webinar Series - Metabolic Disorders

    1:13:43

    The last webinar of our 2021 CAGS Webinar Series was on the topic of Metabolic Disorders. Sponsored by Genpharm, the webinar featured Fathiya Al Murshedi from Sultan Qaboos University and David Kasper from ARCHIMEDlife (ARCHIMED Life Science GmbH), and was moderated by Amal Mohamed Al Tenaiji from Sheikh Khalifa Medical City.

    Register for our upcoming major event, the 9th Pan Arab Human Genetics Conference (cost-free):

    #metabolic #metabolichealth #review #genetics #IEM #metabolism #leukodystrophy #newbornscreening

  • Genetics, Genomics, and Drug Discovery - Lon Cardon

    52:42

    Lecture by Lon Cardon, GlaxoSmithKline, at Target Validation using Genomics and Informatics (8-10 December 2015), organised by the Wellcome Genome Campus Advanced Courses and Scientific Conferences team.

  • GROUP 10 Gene therapy versus stem cell therapy pt.1

    11:11

  • What is the Superhero Vaccine? | The Future of Medicine | Interview | Euan Ashley

    6:40

    A Stanford scientist is developing a 'Superhero vaccine' that will prevent death from heart diseases, strokes, liver problems. The jab will be based on the DNA of Olympic athletes, and will change your genetic make-up forever.

    #EuanAshley #Interview #SuperheroVaccine

    About Channel:

    WION -The World is One News, examines global issues with in-depth analysis. We provide much more than the news of the day. Our aim to empower people to explore their world. With our Global headquarters in New Delhi, we bring you news on the hour, by the hour. We deliver information that is not biased. We are journalists who are neutral to the core and non-partisan when it comes to the politics of the world. People are tired of biased reportage and we stand for a globalised united world. So for us the World is truly One.

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