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AAV Capsid Production with Drs. Mario Mietzsch and Robert McKenna - Molecular Therapy Podcast
Dr. Daniel Stone speaks with Drs. Mario Mietzsch and Robert McKenna from the University of Florida to discuss a recent article published in the Biomanufacturing in Gene and Cell Therapy special issue of Molecular Therapy Methods & Clinical Development (www.sciencedirect.com/special-issue/10RZ8HRVPL2) by Drs. Mietzsch, McKenna, and colleagues titled Production and characterization of an AAV1-VP3-only capsid: An analytical benchmark standard (www.cell.com/molecular-therapy-family/methods/fulltext/S2329-0501(23)00064-5?_returnURL=https%3A%2F%2Flinkinghub.elsevier.com%2Fretrieve%2Fpii%2FS2329050123000645%3Fshowall%3Dtrue) .
If you enjoy today’s conversation, you’ll also enjoy the upcoming ASGCT Policy Summit in Washington, DC, September 23-24.
This can't-miss event brings together policymakers and gene and cell therapy experts, including FDA leaders Julie Tierney and Dr. Nicole Verdun, to discuss the latest policies impacting this rapidly evolving field.
Register now at www.asgct.org/PolicySummit (www.asgct.org/policysummit) for invaluable insights on navigating the regulatory landscape.
In This Episode:
Dr. Daniel Stone
Associate Editor-in-Chief of Molecular Therapy Methods & Clinical Development and Senior Staff Scientist, Infectious Disease Sciences, Vaccine and Infectious Disease Division at Fred Hutch Cancer Center
Dr. Mario Mietzsch
Assistant Scientist, Department of Biochemistry and Molecular Biology, University of Florida
Dr. Robert McKenna
Professor and Director of the Center for Structural Biology, Department of Biochemistry and Molecular Biology, University of Florida
'Electric Dreams' by Scott Buckley - released under CC-BY 4.0.
www.scottbuckley.com.au (protect-us.mimecast.com/s/CXOTCL9w1QUXqP0HBBFDP)
Show your support for ASGCT!: asgct.org/membership/donate
See omnystudio.com/listener (omnystudio.com/listener) for privacy information.
Переглядів: 99

Відео

Gene Insertion Tech with Jason Cole, CEO of SalioGen-ASGCT's The Issue
Переглядів 14414 днів тому
In this Very Special Episode, Emily Walsh Martin, PhD explores the cutting-edge transposase-based technology presented by SalioGen at the ASGCT Annual Meeting in which they achieved gene insertion without double-strand breaks. Join us as Jason Cole, CEO at SalioGen, takes us on a captivating journey through the company's groundbreaking work, including their initial data in Stargardt disease usi...
Meet Dr. Mohamed Abou-el-Enein, Editor-in-Chief of Molecular Therapy Methods & Clinical Developme...
Переглядів 2314 днів тому
Mohamed Abou-el-Enein, MD, PhD, MSPH, the new editor-in-chief of Molecular Therapy Methods & Clinical Development (MTMCD), sits down with Scientific Editor Rory Bricker-Anthony, PhD, to discuss his vision for the journal. If you enjoy today’s conversation, you’ll also enjoy the upcoming ASGCT Policy Summit in Washington, DC, on September 23 and 24 (ASGCT.org/PolicySummit) . This can't-miss even...
Gene Therapy Targets and Biomarkers: Molecular Therapy Presents
Переглядів 381Місяць тому
Develop a comprehensive understanding of target discovery strategies in cancer immunotherapy, the emerging role of tissue-derived extracellular vesicles (Ti-EVs) for disease target identification, and the utilization of biomarkers for lysosomal storage diseases. This event was hosted by ASGCT on April 23, 2024. Enjoy this sample of ASGCT and Molecular Therapy's member-exclusive content, and con...
Increasing Equitable Access with Hayley Lam, PhD
Переглядів 1014 днів тому
At #ASGCT2024, Dr. Lam (CIRM) spoke during a symposium on developing CGTs with a DEI-forward approach. In this interview, she talks about incorporating DEI into the review criteria for CIRM's funded programs, how to measure DEI, and what she'd like to see in the long term for patients. Meeting registrants can watch this session (and all others) through June 21 on the virtual platform (annualmee...
An Explosive Year for Gene Therapy with ASGCT President Paula Cannon
Переглядів 2514 днів тому
As she begins her tenure as ASGCT President, Paula Cannon, PhD (USC), tells host Lynnea Olivarez, incoming chair of the Communications Committee, why she's excited to take the reins for the Society. Show your support for ASGCT!: asgct.org/membership/donate See omnystudio.com/listener (omnystudio.com/listener) for privacy information.
Reporting on Regulatory Considerations with Journalist Jef Akst
Переглядів 714 днів тому
Jef Akst, managing editor at BioSpace, talks about the major CGT issues she's been covering at #ASGCT2024 including regulatory considerations and AAV manufacturing. She also tells host Lynnea Olivarez what she hopes to be reporting on next year at the ASGCT Annual Meeting in New Orleans! Show your support for ASGCT!: asgct.org/membership/donate See omnystudio.com/listener (omnystudio.com/listen...
CNS Gene Therapies with Todd Carter, PhD
Переглядів 1614 днів тому
Todd Carter, PhD (CSO at Voyager Therapeutics) talks with incoming Communications Committee Chair Lynnea Olivarez about changes in the approach to developing gene therapies for CNS disorders like Huntington's disease. Show your support for ASGCT!: asgct.org/membership/donate See omnystudio.com/listener (omnystudio.com/listener) for privacy information.
New Frontiers in Hemophilia Treatments with Lindsey George, MD
Переглядів 1514 днів тому
ASGCT Board of Directors member Lindsey George, MD, (Children's Hospital of Philadelphia) discusses the dramatic advancements in available treatment options for patients with hemophilia and the responsibility of scientists and clinicians in guiding patients through these once-in-a-lifetime decisions. Host: Lynnea Olivarez, incoming chair of the ASGCT Communications Committee Show your support f...
Cystic Fibrosis Gene Therapies with CDA Winner Ashley Cooney, PhD
Переглядів 2214 днів тому
Incoming ASGCT Communications Committee chair Lynnea Olivarez catches up with ASGCT Career Development Award winner Ashley Cooney, PhD, (University of Iowa) to discuss her work in vector-delivered airway gene transfer and use of saline solution to "dramatically increase" gene transfer. Show your support for ASGCT!: asgct.org/membership/donate See omnystudio.com/listener (omnystudio.com/listener...
Patient Advocacy with Leah Myers + Jenny Burke
Переглядів 914 днів тому
Leah Myers, Executive Director of the FamilieSCN2A Foundation, and Jenny Burke, who sits on the organization's board, talk about the importance of empowering patients and families affected by genetic diseases. Host: Lynnea Olivarez Show your support for ASGCT!: asgct.org/membership/donate See omnystudio.com/listener (omnystudio.com/listener) for privacy information.
Stem Cell Therapy with Charles Murry, MD, PhD
Переглядів 1614 днів тому
Dr. Murry talks about his early work reprogramming injured heart cells, his vision for the future of organ regeneration, and the privilege of getting paid to be curious. Host: Lynnea Olivarez Show your support for ASGCT!: asgct.org/membership/donate See omnystudio.com/listener (omnystudio.com/listener) for privacy information.
Communicating the Science with Benjamin McLeod
Переглядів 414 днів тому
"Storytelling is completely underrated in science," says Benjamin McLeod, a lab scientist-turned-communicator. Listen to him talk about the importance of presenting CGT research in a narrative format. Host: Lynnea Olivarez Show your support for ASGCT!: asgct.org/membership/donate See omnystudio.com/listener (omnystudio.com/listener) for privacy information.
New Technologies for Gene Targeting + Gene Correction with David Segal, PhD
Переглядів 1514 днів тому
In this episode, host Lynnea Olivarez talks with David Segal, PhD, an abstract session co-chair at the ASGCT Annual Meeting. Dr. Segal discusses new technologies that he says have moved from academia to industry, and, hopefully soon, to rare disease patients. Miss a session? Catch it on demand on the virtual platform for 30 days following the conclusion of the meeting! Show your support for ASG...
Welcome to the Annual Meeting with ASGCT President Jeff Chamberlain, PhD
Переглядів 122Місяць тому
ASGCT President Dr. Jeff Chamberlain cuts the ribbon at the ASGCT 27th Annual Meeting and, having planned much of this year's event, reflects on nearly three decades of ASGCT. Host: Lynnea Olivarez, ASGCT Communications Committee
ASGCT 2024 Abstract Highlights with Chris Leidli, Emily Walsh Martin, and Lynnea Olivarez
Переглядів 4122 місяці тому
ASGCT 2024 Abstract Highlights with Chris Leidli, Emily Walsh Martin, and Lynnea Olivarez
CMC Strategy with Susan D'Costa, PhD-The Issue
Переглядів 1582 місяці тому
CMC Strategy with Susan D'Costa, PhD-The Issue
Gene Editing Technologies: Transforming Rare Disease Care
Переглядів 1 тис.2 місяці тому
Gene Editing Technologies: Transforming Rare Disease Care
CAR T for Acute Myeloid Leukemia with Dr Marco Davila-Molecular Therapy Podcast
Переглядів 1712 місяці тому
CAR T for Acute Myeloid Leukemia with Dr Marco Davila-Molecular Therapy Podcast
Recent CMC Guidance for Cellular and Gene Therapies: BIO-ASGCT CGT Science Series
Переглядів 1,1 тис.2 місяці тому
Recent CMC Guidance for Cellular and Gene Therapies: BIO-ASGCT CGT Science Series
Around the World: Innovation in Japan
Переглядів 2442 місяці тому
Around the World: Innovation in Japan
Regulatory Landscapes in CGT with Sabrina Mogle-ASGCT's The Issue
Переглядів 1332 місяці тому
Regulatory Landscapes in CGT with Sabrina Mogle-ASGCT's The Issue
Do More in Baltimore - ASGCT's Expanded Exhibit Hall
Переглядів 1893 місяці тому
Do More in Baltimore - ASGCT's Expanded Exhibit Hall
Do More in Baltimore - Attractions
Переглядів 583 місяці тому
Do More in Baltimore - Attractions
Do More in Baltimore - Food
Переглядів 393 місяці тому
Do More in Baltimore - Food
Do More in Baltimore - Explore
Переглядів 633 місяці тому
Do More in Baltimore - Explore
Hot Topics in CGT with WIRED's Emily Mullin: Annual Meeting Preview
Переглядів 823 місяці тому
Hot Topics in CGT with WIRED's Emily Mullin: Annual Meeting Preview
Improving AAV Production with Adam Schieferecke, PhD, + David Schaffer, PhD
Переглядів 2433 місяці тому
Improving AAV Production with Adam Schieferecke, PhD, David Schaffer, PhD
AI in CGT: Previewing the Annual Meeting
Переглядів 713 місяці тому
AI in CGT: Previewing the Annual Meeting
The Issue: AAV Immunogenicity with Federico Mingozzi, PhD
Переглядів 6814 місяці тому
The Issue: AAV Immunogenicity with Federico Mingozzi, PhD

КОМЕНТАРІ

  • @christinarosed.p.1967
    @christinarosed.p.1967 13 днів тому

    1 in 800 will have an adverse event. Responsable for unexpected deaths on a grand scale espessaly using the liponano particles. The long term studies are not completed and unknown. In itself leads to cansers of all kinds. This is a bunch of greedy buggers making money on eugenics, not helping anybody, just a profitable ideoligy that will never work as it in all the years never has not lead to a disaster, Sickning.

  • @Got.A.Ticket_2_Ride
    @Got.A.Ticket_2_Ride Місяць тому

    my baby brother has this. he's only 3. he hasn't left the hospital since he was born. it's been so rough.

  • @yogeshkadyan7792
    @yogeshkadyan7792 Місяць тому

    So sad... Four year old video yet no update anywhere for treatment using gene editing!

  • @danielasutherna8191
    @danielasutherna8191 Місяць тому

    I want to appreciate Dr Madida on UA-cam for treating my Dad of his Multiple sclerosis and parkinson disease with their natural meds💊🌿🌿👌👌..

  • @creativologist1813
    @creativologist1813 Місяць тому

    Why do you say mRNA vaccines for Covid fit YOUR definition of "Gene Therapy?" Elaborate.

  • @fiercetilapia
    @fiercetilapia 2 місяці тому

    beautiful

  • @oefhljfnlj
    @oefhljfnlj 2 місяці тому

    Lecker Lungenbrötchen

  • @seanjohnsovic
    @seanjohnsovic 2 місяці тому

    Grüße an alle

  • @shantakumardhar2391
    @shantakumardhar2391 3 місяці тому

    dear sir or mam, if exon 45,46, and 47 missing how will it reframe it...plz give me suggestions...

  • @JC-ly8pz
    @JC-ly8pz 3 місяці тому

    It would be great if there was a video on how Excision-only PiggyBac Transposase Expression vector works

  • @user-dz1bh2di8q
    @user-dz1bh2di8q 3 місяці тому

    Dr Osade on UA-cam has the best remedy for sickle cell ❤❤❤❤❤❤❤❤

  • @ShilpaSharma-go1xe
    @ShilpaSharma-go1xe 3 місяці тому

    MIMOSA-DI CAPSULES from Planet Ayurveda acts as coolant and useful in blood disorders

  • @chrisbuck6086
    @chrisbuck6086 4 місяці тому

    Any updates on this? I have MTM and would love some updates.

  • @Ivy_1057
    @Ivy_1057 4 місяці тому

    I wonder what is the latest update of this research. Thank you for this presentation!

  • @user-ig5cb9uu9x
    @user-ig5cb9uu9x 4 місяці тому

    Treating disease by changing the blueprint to our bodies at a microscopic level 🧬🩺🥼❤️ (Our Genetic Make-up)

  • @dornie_donko
    @dornie_donko 4 місяці тому

    Eat turd

  • @ladyalexander2003
    @ladyalexander2003 4 місяці тому

    They illicit immune response because it’s all toxic to the human body that is NOT immunity!

  • @GamingPaxels
    @GamingPaxels 5 місяців тому

    This helped me so much! I couldn't understand viral vectors but this video go me to understand.

  • @mathalhaba
    @mathalhaba 5 місяців тому

    شكرا كثيرا 🤍🤍

  • @davidhayes7596
    @davidhayes7596 6 місяців тому

    That's a great report. What's relevant to me is , is that if the gene therapy doesn't work , I can still infuse . Is there any food that that helps. I know personally that sny food that's a blood thinner doesn't help. Vitamin K green type smoothies are safe. It seems like 95 percent of things that are good for blood are risky for bleeders. Thank God for clotting factor. I infuse once a week to control hip both ankles and right knee target joints. Survived a brain aneurysm in 05 . My faith in Jesus got me through that . I'm carb free by about 90 percent of the time. I'm checking into this . This could be wonderful news fot us . And yes I agree that it's unimaginable what people world wide suffer with out clotting factor.

  • @viaocidental8457
    @viaocidental8457 6 місяців тому

    They are using us like laboratory rats. The Covid mRNA genetic experiment, in addition to not working as it should, is causing numerous side effects. Don't believe advertisements with pretty designs because it's not that simple. Big Pharma's goal is money.

  • @user-ne5zc2xc4x
    @user-ne5zc2xc4x 6 місяців тому

    Wow it's very interesting

  • @shekharreddy4683
    @shekharreddy4683 6 місяців тому

    Any cure for tis disease? If anyone knows please please let ne know.thank you

  • @neilmcmanus3727
    @neilmcmanus3727 7 місяців тому

    how long do the EVs persist in the system ? how long can the "exosomes" be detected in the blood system ? therapeutic application dose would be at what numbers/level of "exosomes" ? the medical application requires a single dose ?, or do the doses need to be sequential and at what rate ? how many ? how does one measure the EVs in a 5ml glass vessel ?

  • @ladyalexander2003
    @ladyalexander2003 7 місяців тому

    This hypothesis is toxic! Mrna from any other species isn’t meant to go in the human body along with all these chemicals no wonder people were getting sick after they were injected with this toxic combination claiming it’s a vaccine when it’s actually causing all the diseases to escalate!

  • @jeanieli9999
    @jeanieli9999 7 місяців тому

    Beautiful presentation for beginners learning gene therapy regarding DMD

  • @LiamSturgess
    @LiamSturgess 7 місяців тому

    Funny - "The subtitling of these videos was funded in part by a donation from Pfizer, Inc." Good to know.

  • @yuntingpu327
    @yuntingpu327 7 місяців тому

    Super useful! Thanks for sharing!

  • @zoka843
    @zoka843 7 місяців тому

    Hello, I need to use this medium to thank the Doctor here that I found on UA-cam, I used his herbal medicine and he cured me completely without recurrence👩

  • @edwinmarlongameplays9786
    @edwinmarlongameplays9786 7 місяців тому

    Hello, I need to use this medium to thank the Doctor here that I found on UA-cam, I used his herbal medicine and he cured me completely without recurrence👩

  • @nishargakabir745
    @nishargakabir745 8 місяців тому

    🥰🥰 fell proud as a LGMD patient 🥰🥰

  • @dinaayad4891
    @dinaayad4891 8 місяців тому

    wooooow you are amazing

  • @Mantic0reIlluminati333
    @Mantic0reIlluminati333 8 місяців тому

    So about making me immortal. ...

  • @heidilady
    @heidilady 8 місяців тому

    I have pigment dispersion syndrome. I really need a cure.

  • @islamahmed8924
    @islamahmed8924 8 місяців тому

    😢

  • @readme373
    @readme373 9 місяців тому

    Exciting and well explained

  • @ketannimbalkar9852
    @ketannimbalkar9852 9 місяців тому

    Good elaborate

  • @chadyWinrar
    @chadyWinrar 10 місяців тому

    I know nothing about viral vectors nor any biology really. Just stumbled across the word viral vector and youtubed it. and I am both astonished and greatful for how much science help humanity, and the heroes that dedicate their life to science and research to bring us these amasing treatments.

  • @ladyalexander2003
    @ladyalexander2003 10 місяців тому

    Which species Messenger RNA is this from and what’s the danger of sick animals mRNA controlling human cells? Oh yes zoonotic disease & sickness!

  • @DM-0072
    @DM-0072 10 місяців тому

    I find a high protein diet along with light exercise really helps. Dont excercise till your muscles cramp or get sore...but exercise keeps the muscles strong. Watch your weight but dont get too thin ....more weight = more effort to move. Its a losing battle but try keep mobile and safe as long as you can

  • @junkman7901
    @junkman7901 10 місяців тому

    Gene therapy

  • @junkman7901
    @junkman7901 10 місяців тому

    It’s in the jab

  • @imopman
    @imopman 10 місяців тому

    Look, I feel sorry about those suffering from this but since this IS a genetically inherited disease then along with treatment should also come sterilization. Why create new generations of people with this? Adopt a healthy child instead.

  • @nawrasaqidi5885
    @nawrasaqidi5885 10 місяців тому

    THANK YOU SO MUCH

  • @carterantwuan7369
    @carterantwuan7369 11 місяців тому

    Today i am free from herpes with the help of DR. harry herbs on UA-cam who cure me from herpes virus with his Herbal medicine.

  • @jasonichigo306
    @jasonichigo306 11 місяців тому

    Immortality

  • @ayliinayc5909
    @ayliinayc5909 Рік тому

    Hi. Iam a 29 years old women and i havemyotubular myopathy, i hope for a cure. Do you have an e mail adress?i want to ask some questions or more information,

  • @juno6
    @juno6 Рік тому

    Poison.

  • @jalalshahshah546
    @jalalshahshah546 Рік тому

    Gene therapy is started any where in world?

  • @maryamtanoli
    @maryamtanoli Рік тому

    Hi my elder brother is suffering from eb since 30 years there is no cure we tried everything literally everything my cousin was also butterfly baby but he was died in 13 years of his age