Publications

Peer Reviewed Work Supporting Our Therapeutic Approach

In vivo gene correction

Wang, L. et al.  Meganuclease targeting of PCSK9 in macaque liver leads to stable reduction in serum cholesterol. Nat Biotechnol. 8, 717-725 (2018).

-Press release from Penn Medicine

-Write up in Science

Allogeneic CAR T

McCreedy, B. J. et al. Off the shelf T cell therapies for hematologic malignancies. Best Pract Res Clin Haematol. 2, 166-175 (2018).

Macleod, D.T. et al.  Integration of a CD19 CAR into the TCR alpha chain locus streamlines production of allogeneic gene-edited CAR T cells. Mol Ther. 4, 949-961 (2017)PDF