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.
The CMTRF awarded a $500,000 grant to Afrooz Rashnonejad, PhD, Assistant Professor at Ohio State University's Department of Pediatrics and Principal Investigator at the Nationwide Children's Hospital Center for Gene Therapy, in Columbus, Ohio.
Read more: cmtrf.org/nationwide-childre…
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' theconversation.com/how-robe… 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: insideprecisionmedicine.com/…
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)
#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 nature.com/articles/s41594-0…
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: nature.com/articles/s41434-0…
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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. sciencedaily.com/releases/20…
Excited that our work with the lab of Ruth Brack-Werner is now out in Advanced Science: onlinelibrary.wiley.com/doi/… 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 👏🥂 !!
A new #HIV clinical trial shows promising results using a "strategy involving an adeno-associated viral vector-based gene delivery system that instructs cells to pump out antibodies that block HIV." bit.ly/38JNBgB via @sciencedaily
Gene&Cell Therapy >> Abzena commits chunk of new $65M investment to build out in hot North Carolina scene: A San Diego CDMO has landed a $65 million investment to expand its drug substance and fill-finish… dlvr.it/SNSCQP#lucidquest#genetherapy#celltherapy
In a recent paper, researchers demonstrated the long-term safety and effectiveness of #genetherapy to treat #WiskottAldrich syndrome. Up to 9 years after treatment, no adverse effects, and patients had dramatic symptom improvement: bddy.me/3jHwUEV