Matthew L. Hirsch

TL;DR: It is demonstrated that anti-CD19 CAR T cells produced in this manner do not express the endogenous TCR, exhibit potent effector functions in vitro, and mediate clearance of CD19+ tumors in an in vivo mouse model.

TL;DR: The transduction efficiency of ten different AAV serotypes in thirty-four different mammalian cell lines and primary cell types is described to provide an important and unique resource for investigators who use AAV as an ex vivo gene delivery vector or who work with cells that are difficult to transfect.

TL;DR: The data provide the basis for increased retrograde transduction efficiency using peripheral injections of scAAV1 vectors for therapeutic gene delivery to the spinal cord.

TL;DR: Demonstration of long-term phenotypic correction of hemophilia A dogs with combination adjuvant bortezomib and AAV vector expressing the oversized transgene establishes preclinical studies that support testing in humans and provides a working paradigm to facilitate a significant expansion of therapeutic targets for human gene therapy.

TL;DR: The ability to deliver ZFNs and a repair substrate by a single AAV vector is demonstrated and insights for the optimization of rAAV-mediated gene correction using drug therapy are offered.