Somatic Cell Genome Editing (SCGE) Outreach @somaticediting
The Somatic Cell Genome Editing Consortium is funded by the @NIH_CommonFund. Retweets ≠ endorsements. Visit the SCGE Toolkit https://t.co/40whIufXjO scge.mcw.edu Joined September 2019-
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SCGE researchers demonstrate proof-of-concept of multiorgan mutation correction in cystic fibrosis using peptide nucleic acids encapsulated in polymeric nanoparticles and delivered systemically in utero. #PublicationFriday pnas.org/doi/10.1073/pn…
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The SCGE Gene Therapy Clinical Trial Browser has many use cases for researchers, clinicians, patients, and more. Click through these images for examples of how different individuals could use the browser. To start your own search, go to scge.mcw.edu/platform/data/…
This review highlights next-generation technologies under development to expand the reach of gene therapies by enabling precise modulation of gene expression. #PublicationFriday sciencedirect.com/science/articl…
Congrats to SCGE Researchers Jennifer Doudna and David R. Liu for receiving 2026 National Awards from the American Chemical Society! Dr. Doudna is the recipient of the Priestley Medal, while Dr. Liu received the ACS Award for Creative Invention. cen.acs.org/people/awards/…
Somatic Cell Genome Editing (SCGE) Outreach retweeted
Please join us in congratulating HHMI Investigators Jennifer Doudna and David R. Liu (@davidrliu), recipients of national awards from the American Chemical Society (@AmerChemSociety) for 2026. 🔗cen.acs.org/people/awards/…
SCGE researchers report the first application of base editors in primary natural killer cells to achieve both loss-of-function and gain-of-function mutations. #PublicationFriday jitc.bmj.com/content/13/5/e…
Somatic Cell Genome Editing (SCGE) Outreach retweeted
CRISPR with a dimmer switch. Cas9-degron + FDA-approved pomalidomide = precise, reversible control of genome editing in human cells. @somaticediting @NIH doi.org/10.1016/j.omtn…
Here SCGE researchers used cytosine and adenine base editing to reduce the repetitiveness of TNRs in patient cells and in mice, demonstrating that introducing interruptions in pathogenic TNRs can mitigate a key neurological feature of TNR diseases in vivo. nature.com/articles/s4158…
Congrats to Rebecca Ahrens-Nicklas for being named Rad Girl of the Year at the 2025 Rad Awards! Dr. Ahrens-Nicklas was nominated for her groundbreaking work in personalized gene therapy with baby KJ. Learn more about the 2025 Rad Award winners at thephiladelphiacitizen.org/2025-rad-award…
SCGE researchers report the development of elastin-based nanoparticles for therapeutic delivery (ENTER), a recombinant elastin-like polypeptide (ELP)-based delivery system for effective cytosolic delivery of biomacromolecules in vitro and in vivo. nature.com/articles/s4158…
In this study, researchers introduce a method for high-purity AAV vector production utilizing recombination-dependent minicircle formation and genetic coupling (AAVPureMfg). #PublicationFriday embopress.org/doi/full/10.10…
Somatic Cell Genome Editing (SCGE) Outreach retweeted
Today in @CellCellPress we report the use of prime editing to correct several mutations that cause alternating hemiplegia of childhood (AHC), a rare and devastating neurodevelopmental disorder, in patient-derived cells and in two mouse models. drive.google.com/file/d/1ibC50t… 1/10
SCGE researchers develop a base editing approach exploiting a naturally occurring CD33 single nucleotide polymorphism leading to removal of full-length CD33 surface expression on edited cells. #PublicationFriday nature.com/articles/s4146…
Here researchers use single-molecule torque spectroscopy to investigate the dynamics and mechanics of R-loop formation of two widely used Cas12a orthologs at base-pair resolution. #PublicationFriday nature.com/articles/s4146…
Gene Therapy News: Plans for the Center for Pediatric CRISPR Cures at the University of California San Francisco were announced today (July 8th). The Center is a collaboration between the Innovative Genomics Institute and the Chan Zuckerberg Initiative. time.com/7300257/center…
Somatic Cell Genome Editing (SCGE) Outreach retweeted
The Phase 2 winners of the TARGETED Challenge were announced! The TARGETED Challenge was designed to advance the cutting-edge research of targeted delivery systems for genome editors in somatic cells. Learn more about the winners: go.nih.gov/395Z6l4 #GenomeEditing
Here, a 3D tissue-engineered model of human skeletal muscle ("myobundles") is utilized as an in vitro platform for temporal control and studies of Notch signaling. #PublicationFriday advanced.onlinelibrary.wiley.com/doi/10.1002/ad…
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