Congratulations to ASGCT Associate Member @rashnonejad, who received a grant to help develop a #genetherapy for Charcot-Marie-Tooth disease type 1B (#CMT1B)! https://t.co/PakImQgp9P
@KeithNFargo@OhioState@nationwidekids I am grateful for partnering with CMTRF and hopeful that together we can make a difference for people with CMT1B.
Congratulations to Afrooz @Rashnonejad of @OhioState and @NationwideKids! This is a very promising project aiming to develop a single gene therapy that has the potential to work for all of nearly all people with CMT1B.
American Society of Gene and Cell Therapy (ASGCT), the best place to meet colleagues and discuss science. There was a great discussion after my talk. Thanks to all organizers.
Come to connect if you are here!
#asgct22#asgct#meeting#talk#neuromuscular#FSHD#CRISPR
How Robert Langer, a pioneer in delivering mRNA into the body, failed repeatedly but kept going: 'They said I should give up, but I don't like to give up' https://t.co/D7PWkTID42 via @ConversationUS
Five RNA Therapeutics Startups to Watch in 2022
Five RNA therapeutics startups to watch that have opened their doors since 2020 and are developing the next generation of RNA medicines. Learn more: https://t.co/iQ8fFfMVqt
On this #NationalDNADay, we celebrate Dr. Rosalind Franklin. She discovered the density and helical form of DNA but did not receive the
📷: Rosalind Franklin with microscope in 1955. Author: MRC Laboratory of Molecular Biology. (CC-BY-SA-4.0)
New IPOPI publication - leaflet on WHIM syndrome!
#WHIMsyndrome is a rare genetic #primaryimmunodeficiency with very specific charateristics - read all about it and share with your local network
https://t.co/G5PDmdnRoW
#CRISPR – #Cas9 bends and twists DNA to read its sequence and locate target sites. Read more about how the @doudna_lab and collaborators made this discovery 🧬
Nature Structural & Molecular Biology https://t.co/DKkBFA30LJ
To comprehensively mitigate the misuse of synthetic viruses, we need to consider accessible & powerful methods arising from viral vector engineering for gene therapy. In a new paper in @GeneTherapy_SN, we discuss these risks and their mitigation: https://t.co/d3xQBozSld
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New approach for delivery of anti-HIV antibody therapy shows promise in phase I clinical trial: Treatment using adeno-associated viral vector is poised to move into larger, later-phase trials. https://t.co/mDcM2Sybwc
Excited that our work with the lab of Ruth Brack-Werner is now out in Advanced Science: https://t.co/6AMUjgg0Th We identified the receptor signature of astrocytes for an RGD-displaying AAV9 incl integrins & AAVR. Congrats Jad from our lab, Amelie (1st), Ruth & all others 👏🥂 !!