Prof David J. Segal

Experts / Prof David J. Segal

Prof David J. Segal

Biochemistry and Molecular Medicine, Pharmacology, Genome Center, MIND Institute; Co-Chair, Integrative Genetics and Genomics graduate group; Field Chief Editor, Frontiers in Genome Editing

Dr. Segal’s research, together with the labs of Kyle Fink and Jill Silverman, focuses on developing molecular therapies for neurologic disorders. We use programable DNA- or RNA-binding platforms (Zinc finger, TALE, CRISPR/Cas9, Cas12, Cas13) to cause long-term changes in the expression of disease genes. This approach avoids problematic double-strand breaks. We use a variety of delivery systems (protein, AAV, lipo-particles, stem cells) to treat the brain in animal models of disease, which are extensively characterized on a molecular and behavioral level. A flagship project is Angelman syndrome, for which the therapeutic strategy is to reactivate a silenced gene in the brain. We collaborate with groups around the world to de-risk genetic therapies for this and several other related disorders.

Angelman Syndrome – Gene therapy

Prof David Segal discusses gene therapy, how it works, conditions which gene therapy is currently approved, and future direction of gene therapy in the potential treatment of Angelman Syndrome.

Angelman Syndrome – Gene therapy

Prof David Segal discusses gene therapy, how it works, conditions which gene therapy is currently approved, and future direction of gene therapy in the potential treatment of Angelman Syndrome.

By Prof David J. Segal