Biomedical Engineering

Therapeutic genome editing using CRISPR-Cas9

January 17, 2017

4:30 a.m. ET

A302 Doherty Hall

Thomas Gaj, Ph.D.

Postdoctoral Fellow
Department of Bioengineering
University of California

Abstract

Neurodegenerative disorders affect over 30 million people worldwide and are the leading cause of disability in the aging population. In recent years, considerable progress has been made in unraveling the genetic etiology of neurodegeneration, offering new hope that targeted therapeutics can be developed to treat these devastating disorders. The past decade has also witnessed the emergence of several genome editing technologies, including CRISPR-Cas9, which have endowed investigators with the ability to directly modify disease-associated genomic loci. We have established an approach to treat neurodegeneration by in vivo genome editing that effectively delays disease onset, improves motor function, reduces muscle atrophy, and importantly, extends survival in mouse models of these diseases. These findings demonstrate the potential of genome editing to treat disorders of the central nervous system.

Coffee and snacks are served at 4:00 p.m. in Scott Hall BME kitchen area, where students may meet the speaker.

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