Somatic Cell Genome Editing Consortium

The SCGE has recently released a Gene Therapy Clinical Trial Browser. This publicly accessible, free database was created for the benefit of users seeking information on gene therapy development. The information within integrates various sources - including clinicaltrials.gov, publications, sponsor press releases, presentations, and more - to give a comprehensive overview of the gene therapy clinical trial landscape. Check out the browser at: https://lnkd.in/gahu3Kmy #clinicaltrials #genetherapy #research

A team of SCGE researchers used structural and biochemical methods to assess early steps in DNA recognition by Cas12a protein-guide RNA complexes. They show here that Cas12a initiates DNA target recognition by bending DNA to induce transient nucleotide flipping that exposes nucleobases for DNA-RNA hybridization. Their findings support a model in which RNA-mediated DNA interference begins with local helix distortion by transient CRISPR-Cas protein binding. https://lnkd.in/ddVvG9XQ #RNA #CRISPR #DNA #publication

This SCGE project aims to develop a one-time therapy for prion disease by targeting the single genetic root cause of disease. The project is lead by Sonia Vallabh, Eric Vallabh Minikel, Ben Deverman, David R. Liu, and Jonathan Weissman. https://lnkd.in/ez52b33G #raredisease #research #genetherapy

?? Join us for Empowering Patients 2025: A Cell and Gene Therapies Summit on March 12-13! "Patients and advocates play such an important role as partners in developing cell and gene therapies. You raise funds for research, collate data about your conditions, lobby, educate your communities, and generously participate in clinical trials." - Dr. Paula Cannon, ASGCT President This FREE virtual event brings together experts, advocates, and clinicians to discuss the unique challenges and possibilities of cell and gene therapies. Learn about: - The science behind treatment approaches - Navigating complex clinical trial design - Managing expectations during the therapy journey - Solutions when innovation faces obstacles As Dr. Cannon shares: "The insights I've gained from patient communities have made me braver and more creative as a scientist. It's given me a sense of urgency and is a major reason I feel inspired and honored to be part of this work." Register now for this inaugural summit dedicated to empowering patients, advocates, and caregivers in the CGT space: www.asgct.org/ep2025 #EmpoweringPatients2025 #CellAndGeneTherapy #RareDiseaseAdvocacy #ASGCT

A team of SCGE researchers present GARNET (Gtdb Acquired RNa with Environmental Temperatures), a new database for RNA structural and functional analysis anchored to the Genome Taxonomy Database (GTDB). GARNET links RNA sequences to experimental and predicted optimal growth temperatures of GTDB reference organisms. Using GARNET, the team develops sequence- and structure-aware RNA generative models, with overlapping triplet tokenization providing optimal encoding for a GPT-like model. Leveraging hyperthermophilic RNAs in GARNET and these RNA generative models, they identify mutations in ribosomal RNA that confer increased thermostability to the Escherichia coli ribosome. The GTDB-derived data and deep learning models presented here provide a foundation for understanding the connections between RNA sequence, structure, and function. https://lnkd.in/dHxSdA3r #RNA #genetherapy #models #science

This SCGE project aims to optimize and qualify both CHANGE-seq and gRNA sequencing methods for use as genome editing Investigational New Drug (IND)-enabling assays for regulatory submissions. The project is lead by Shengdar Q. Tsai. https://lnkd.in/e8CP4ZGW #research #genetherapy #regulatory

In this review, SCGE researchers resolve seemingly conflicting findings by considering the different transepithelial voltages and resultant movement of Cl? during short-circuit and physiological open-circuit conditions. This analysis indicates that behavior under short-circuit conditions cannot be directly extrapolated to infer behavior under physiologic conditions. They also discuss the potential role for basolateral Cl? channels in controlling absorption and secretion in ionocytes. https://lnkd.in/dQCQAVck #publication #science

This SCGE project aims to use novel non-viral delivery systems to target the retina efficiently and treat rare conditions like Best Disease and Leber Congenital Amaurosis. The project is lead by Krishanu Saha. https://lnkd.in/eRn8e2v7 #research #genetherapy #raredisease

The SCGE has recently released a Gene Therapy Clinical Trial Browser. This publicly accessible, free database was created for the benefit of users seeking information on gene therapy development. The information within integrates various sources - including clinicaltrials.gov, publications, sponsor press releases, presentations, and more - to give a comprehensive overview of the gene therapy clinical trial landscape. Check out the browser at: https://lnkd.in/gahu3Kmy #clinicaltrials #genetherapy #research

This SCGE project, lead by Bill Peranteau and Kiran Musunuru, aims to use a base editing to directly and permanently correct gene mutations or otherwise alter genes at the DNA level in the liver for patients with phenylketonuria (PKU), hereditary tyrosinemia type 1 (HT1), and mucopolysaccharidosis type 1 (MPSI). https://lnkd.in/ggY-MMKK #raredisease #research #baseediting #genetherapy