Somatic Cell Genome Editing (SCGE) Outreach

A team, including SCGE researchers, conducted an exploratory interview study of Swiss-based perspectives on the US FDA's platform technology designation program to assess the acceptability and feasibility of a similar program in Europe. #PublicationFriday link.springer.com/article/10.118…

Dr. Mohl is a Postdoctoral Scholar at the University of California, San Francisco. He is part of the SCGE project titled “Correction of Neurological Disease via Allele Specific Excision of Pathogenic Repeats”.

In this interview, Greg Mohl, PhD highlights the challenges of delivering CRISPR therapies to the brain and discusses how he and the research team try to balance designing the most effective therapy with the time pressure to get therapies to patients. youtube.com/watch?v=QGQdVn…

This review provides an overview of the current state and future directions of CRISPR-based gene editing in nephrology, underscoring its transformative potential in advancing kidney disease research and therapeutics. #PublicationFriday academic.oup.com/ckj/article/18…

The FDA extended approval of the first gene therapy for sickle cell disease to patients ages 2+, an important public health milestone—expanding access to innovative treatment options for a serious condition that affects thousands of Americans. fda.gov/news-events/pr…

Jennifer is the COO at AS2 Bio Inc and the Head of Regulatory & Policy at Foundation for Angelman Syndrome Therapeutics (FAST). She is part of the SCGE project titled "A Non-viral CRISPR-mediated Genome Editing Delivery Platform As A Potential Therapy For Neurogenetic Diseases".

In this interview, Jennifer Panagoulias provides her perspective on some current regulatory frameworks, and shares practical advice for academic labs preparing their first IND application for a gene therapy. Watch the full interview at: youtube.com/watch?v=53WPxe…

In this study, SCGE researchers isolated a library of natural capsids from human tissues and identified two variants that conferred strong transduction of retinal tissues following intravitreal injections into mice and non-human primates. sciencedirect.com/science/articl…

📢 Registration and abstract submissions are officially open for Breakthroughs in Cell & Gene Therapy! Explore our 2026 program tracks, view abstract categories, and secure your spot today: asgct.org/breakthroughs

We're excited to share the third edition of the SCGE Spotlight! Read the newsletter at linkedin.com/pulse/scge-spo…

Great honor to receive this Outstanding Publication in Basic Research Award from our college UF COP. Our research lab published more than 10 journal papers in this fiscal year, showing our passion and dedication to R & D for finding the best cure ✊

A team of researchers employed tumor-tropic human mesenchymal stem cells as delivery vehicles for magnetotheranostic gold-iron oxide nanoflowers (GIONF), enabling widespread i.t. distribution and sustained retention in contrast to injection of naked GIONF. pmc.ncbi.nlm.nih.gov/articles/PMC12…

Industry News: Beam Therapeutics Announces Clearance of Investigational New Drug Application for BEAM-304 for the Treatment of Phenylketonuria (PKU) by the United States (U.S.) Food and Drug Administration investors.beamtx.com/news-releases/…

Dr. Clark is a Research Scientist at the Medical College of Wisconsin, and is part of the SCGE Translational Coordination and Dissemination Center (TCDC). Find the free resources mentioned in the video at scge.mcw.edu/platform/home

In this "Meet the Researcher Interview", hear from Karen Clark, PhD, as she highlights the free resources available on the SCGE platform. youtube.com/watch?v=O2gjl0…

Today we provided a regulatory update on a path forward for AMT-130 for #huntingtonsdisease. Read the full update: uniqure.gcs-web.com/news-releases/…

In 3 new studies, @davidrliu's lab has optimized key prime editing components (guide pegRNA motifs, reverse transcriptase stability and delivery via lipid nanoparticles), addressing bottlenecks that previously impeded its use in animals and human patients. broad.io/PrimeEditing3

FDA is accepting applications until June 30, 2026, for the Rare Disease Endpoint Advancement (RDEA) Pilot Program, which supports novel efficacy endpoint development for drugs that treat rare diseases. Learn more & submit your application today ➡️ fda.gov/drugs/developm…

Today in @NatureNano, we report an all-RNA lipid nanoparticle (LNP) system for efficient in vivo prime editing (PE). We identify bottlenecks in transient prime editor delivery, develop a workflow for LNP optimization, and use it to rescue a mouse model of phenylketonuria. drive.google.com/file/d/1-rPAaa… 1/12

CRISPR-based gene editing therapies are entering the clinic, offering transformative potential but also presenting potential risks. Preclinical-to-clinical toolkits are needed to assess safety and efficacy of these new therapies and accelerate progress. nature.com/articles/s4157…