Leland Stanford Junior University

Boco Silicon Valley, Inc.

Daniel P Dever

Matthew Hebden Porteus

Nobuko Uchida

Rasmus O Bak

Ann Tsukamoto

Ayal Hendel

Waracharee Srifa

Joseph T Clark

Eric J Kildebeck

In certain aspects, the present invention provides methods for inducing a stable gene modification of a target nucleic acid via homologous recombination in a primary cell, such as a primary blood cell and/or a primary mesenchymal cell. In certain other aspects, the present invention provides methods for enriching a population of genetically modified primary cells having targeted integration at a target nucleic acid. The methods of the present invention rely on the introduction of a DNA nuclease such as a Cas polypeptide and a homologous donor adeno-associated viral (AAV) vector into the primary cell to mediate targeted integration of the target nucleic acid. Also provided herein are methods for preventing or treating a disease in a subject in need thereof by administering to the subject any of the genetically modified primary cells or pharmaceutical compositions described herein to prevent the disease or ameliorate one or more symptoms of the disease.

Pharmaceutical compositions comprising gene-corrected primary cells

Nuclease-mediated genome editing of primary cells

Nuclease-mediated genome editing of primary cells and enrichment thereof

Growing community of inventors

Stanford, CA, United States of America

Daniel P Dever