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Hear from SCGE researcher Ben Deverman this morning as he presents during the Outstanding New Investigator Symposium at #ASGCT2025.

Hear from Ben Deverman as he gives his presentation titled "Designing wet lab experiments to leverage ML". #ASGCT2025

Check out this talk from Xiao Wang, "Corrective Editing to Treat Pseudoxanthoma Elasticum". #ASGCT2025

ICYMI: Congrats to the SCGE researchers working on this groundbreaking therapy! nih.gov/news-events/ne…

Last day of #ASGCT2025! Hear from Varun Katta, who's giving a talk titled: "CHANGE-seq-BE Enables Simultaneously Sensitive and Unbiased In vitro Profiling of Base Editor Genome-Wide Activity".

SCGE researcher Azusa Matsubara is presenting at #ASGCT2025 this morning: "Pooled GUIDE-seq-2 enables simultaneous assessment of cellular activity and specificity for thousands of Cas9 targets".

Hear from Simon Pacouret this morning as he gives a talk titled "Antibody-evading AAV capsids compatible with CNS and muscle-targeting modifications". #ASGCT2025

Another medical milestone! The first patient treated with a prime edited therapeutic shows positive clinical outcomes consistent with effective rescue of his chronic granulomatous disease (CGD), a debilitating and life-shortening primary immunodeficiency. At least 19 base editing

#NIH funded researchers from Children's Hospital and Penn Medicine have treated a child born with a #RareDisease with newly developed #GeneTherapy technology. Learn more: go.nih.gov/vo4Tnf0

The NIH has announced Phase 2 winners of the $6M TARGETED Challenge, advancing in vivo delivery systems for #genome editing. Winning teams developed programmable delivery platforms and technologies to cross the blood-brain barrier. Learn more: freelancer.com/nih/targeted-c… #prize

Excited to be part of the Challenge Team under Somatic Cell Genome Editing (SCGE) Outreach and partnering with Freelancer. Here is looking towards Phase 3! #geneediting #genetherapy #genedelivery

Nature Biotechnology Research Briefing Engineering non-viral protein nanoparticles to deliver nucleic acids and proteins to cells rdcu.be/emZlx Elastin-based nanoparticles for therapeutic delivery Somatic Cell Genome Editing (SCGE) Outreach

SCGE researchers recently published an article on peptide-enabled ribonucleoprotein delivery for CRISPR engineering (PERC), a new approach for ex vivo genome editing of primary human cells. #PublicationFriday nature.com/articles/s4159…

Gene Therapy News: Research teams funded by the NIH have created a set of gene delivery systems that can reach different neural cell types in the human brain and spinal cord with exceptional accuracy. Learn more about the toolkit: nih.gov/news-events/ne…

.Jason Mast provides a clear-eyed summary of how we are getting from N=1 (Baby KJ) to N=many. STAT statnews.com/2025/05/26/wha…

In Nature Genetics we report that base editing of trinucleotide repeats (TNRs) reduces somatic repeat expansions in Huntington’s disease (HD) and Friedreich’s ataxia (FRDA)—in patient-derived cells and in vivo—a collaboration with the Mouro Pinto lab. drive.google.com/file/d/1on7kdy… 1/14

Thrilled to highlight the brilliant work by Mandana Arbab, Żaneta Matuszek, Ricardo Mouro Pinto, David R. Liu, and the team — another major leap forward from Somatic Cell Genome Editing (SCGE) Outreach. 👏🧬 Base editing as a therapeutic strategy for repeat expansion diseases Nature Genetics nature.com/articles/s4158……

This article, with input from SCGE researchers, reviews the application of both 2D and 3D microscopy approaches for monitoring and studying 3D cell cultures. #PublicationFriday nature.com/articles/s4159…