ASGCT (@ASGCTherapy) / X

ASGCT

8,675 posts

ASGCT

@ASGCTherapy

Advancing knowledge, awareness, and education leading to the discovery and clinical application of genetic and cellular therapies to alleviate human disease.

Joined January 2013

ASGCT
@ASGCTherapy
Nov 3, 2021
Replying to @rosenbusch_ @MintNewbee and 3 others
Hi, ASGCT's definition of gene therapy is the introduction, removal or change in genetic material—DNA or RNA—into the cells of a patient to treat a specific disease. Per that definition, the vaccines are gene therapy.
ASGCT
@ASGCTherapy
May 17, 2023
.@JKeithJoung receives the Outstanding Achievement Award for his work in #geneediting! Congrats, Dr. Joung! #ASGCT23
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ASGCT
@ASGCTherapy
May 11, 2024
Wow, what a week at #ASGCT2024! We hope you learned from the science, met fellow members, and saw some great presentations this week! THANK YOU, everyone! See you in New Orleans next year!
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ASGCT
@ASGCTherapy
May 24, 2019
#Zolgensma is now both the second-ever AAV-delivered #GeneTherapy approved for clinical use in the US and the second treatment approved for SMA. Congratulations to all the scientists, clinicians, and patients involved in this landmark achievement. More from ASGCT to come.
FDA Commissioner
@FDACommissioner
May 24, 2019
Today, @US_FDA approved a gene therapy to treat children less than two years of age with spinal muscular atrophy (SMA), the most severe form of SMA and a leading genetic cause of infant mortality. go.usa.gov/xm738
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ASGCT
@ASGCTherapy
Mar 10, 2021
"Mavis was not only a world class scientist, but her character and passion were also unparalleled." Read this tribute to Mavis Agbandje-McKenna, Ph.D. (@UF), the recipient of our 2020 Outstanding Achievement Award. bit.ly/3tarXay
ASGCT
@ASGCTherapy
May 17, 2023
David Liu, PhD, (@HarvardU) speaking now on #baseediting + #primeediting at #ASGCT23!
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ASGCT
@ASGCTherapy
May 16, 2022
Good morning #ASGCT22 and welcome to day one! Today: Visit the Exhibit Hall and learn from education sessions, scientific symposia, and the first set of oral abstracts presentations. More info on our blog: bit.ly/3Mi5UZt
ASGCT
@ASGCTherapy
May 19, 2022
That's a wrap, #ASGCT22! We hope you had an informative, fun week reconnecting with old colleagues, meeting new people, asking questions, and learning about #genetherapy & #celltherapy! Thank you for joining us & catch sessions on demand for 30 days! bit.ly/3lnY5FC
ASGCT
@ASGCTherapy
May 15, 2025
Incredible story that we learned about today at #ASGCT2025!
Children's Hospital
@ChildrensPhila
May 15, 2025
CHOP & @PennMedicine have delivered the first-ever personalized gene editing therapy for a patient with CPS1 deficiency, marking a major milestone in the application of CRISPR-based treatments. Learn more about KJ & the future of personalized medicine: ms.spr.ly/6015SZLAx.
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ASGCT
@ASGCTherapy
Jun 29, 2023
Today, #FDA approved the first #genetherapy to treat adults with severe Hemophilia A, #Roctavian. Approximately 1 in 10,000 people have the blood clotting disorder. Read the statement from ASGCT President Jeffrey Chamberlain, PhD (@UW): asgct.org/publications/n…
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ASGCT
@ASGCTherapy
Nov 3, 2025
📢 AVAILABLE NOW: Breakthroughs in Targeted In Vivo Gene Editing abstracts! Explore the latest gene editing research from leading scientists and clinicians. View and download the collection of original research on our website, and don't miss your chance to dive deeper at the
ASGCT | Breakthroughs in Targeted In Vivo Gene Editing
From asgct.org
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ASGCT
@ASGCTherapy
Jan 11, 2023
David R. Liu @davidrliu, PhD, from @broadinstitute will speak on #BaseEditing and #PrimeEditing at the ASGCT Annual Meeting #ASGCT23 in person on May 17. Dr. Liu’s group pioneered these topics. Register with early bird pricing today annualmeeting.asgct.org/program/plenar…
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ASGCT
@ASGCTherapy
Apr 1, 2020
ASGCT has come to the conclusion that it is only possible to fulfill the Society’s mission by hosting #ASGCT20 virtually. More information is available here: bit.ly/2RbpJb5
ASGCT
@ASGCTherapy
Jul 5, 2018
Following treatment in July 2016 at UCLA, a young Lebanese boy who has been cured of severe combined immunodeficiency #BubbleBoy disease. 30 of 30 patients have been cured in UCLA’s six trials run by ASGCT member, Dr. Donald Kohn.
UCLA doctor credited with curing child with bubble baby disease
From whittierdailynews.com