Project: A Novel Human T-Cell Platform to Define Biological Effects of Genome Editing

PI:   Shengdar Q Tsai, PhD 

Genome editing technologies have extraordinary potential as the basis of new genomic medicines that address the underlying genetic causes of human disease; however, it is challenging to predict their long-term safety, because we do not know the consequences of potential unintended side effects of genome editing such as off-target mutations or immunogenicity. To define the biological effects of genome editing strategies, we will develop a human primary T-cell platform to sensitively detect functional effects coupled with an empirically-trained artificial intelligence models to predict them. Together, our platform will significantly improve confidence in safety assessments of promising genome editing therapeutics.

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