Paula Cannon, PhD


Paula Cannon is a professor at the Keck School of Medicine of the University of Southern California. She studies the genetic modification of hematopoietic stem cells (HSC) to treat HIV infection and genetic diseases, where she is especially known for her work using gene editing technologies such as zinc finger nucleases (ZFNs) and CRISPR/Cas9.

Her group has made a number of technical advances that have accelerated their application, including the demonstration that ZFNs could precisely knock out the CCR5 gene in HSC, and thereby produce an anti-HIV effect. These findings led to funding from the California Institute of Regenerative Medicine to develop a clinical trial in HIV-infected individuals. More recently, she has described a platform using AAV vectors in HSC that allows broader applications of gene editing, beyond gene knock-out. Finally, she is using humanized mouse models in several ongoing collaborations, including the evaluation of therapies for neurological diseases.

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ASGCT Policy Summit

September 23-25, 2020