Abstract

Engineering Genetic Cures: Genome Editing of Stem Cells

Edward Lanphier, CEO, Sangamo Biosciences Inc

Advances in human hematopoietic stem cell (hHSC) isolation, purification and re-engraftment combined with the ability to engineer zinc finger DNA binding protein nucleases (ZFNs) enabling the targeted and permanent modification of any investigator chosen gene sequence have combined to create therapeutic outcomes capable of generating lifelong gene tic cures. This approach is being applied in HIV (targeted disruption of the CCR5 gene in HSCs) and in beta-thalassemia and sickle cell disease (targeted disruption of BCL11A). However, the generality of the ZFN platform permits the target disruption and targeted gene correction of any therapeutically relevant gene thus providing a platform for engineering genetic cures through genome editing of stem cells.