Publications

See below for our latest publications and poster presentations.

Allogeneic CAR T

Shah, B.D. et al. Preliminary safety and efficacy of PBCAR0191, an allogeneic, off-the-shelf CD19-targeting CAR-T product, in relapsed/refractory (r/r) CD19+ NHL. American Society of Clinical Oncology 2021 Annual Meeting.

Jacobson C.A. et al. Initial Findings of the Phase 1 Trial of PBCAR0191, a CD19 Targeted Allogeneic CAR-T Cell Therapy. American Society of Hematology 2019 Annual Meeting.

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

 

In Vivo Gene Correction

Zekonyte, U. et al. Mitochondrial targeted meganuclease as a platform to eliminate mutant mtDNA in vivo. Nat Commun. 12, 3210 (2021).

Jalligampala, A. et al. Rho 1-2 meganuclease, an allele-specific gene-editing therapy, rejuvenates rod photoreceptor structure and function in a pig model of autosomal dominant Retinitis Pigmentosa (adRP). Association for Research in Vision & Ophthalmology 2021 Annual Meeting.

Greig, J.A. et al. Translation of an AAV-delivered gene editing approach for transthyretin amyloidosis in animal models. American Society of Gene and Cell Therapy 2021 Annual Meeting.

Wang, L. et al. Long-term stable reduction of low-density lipoprotein in nonhuman primates following in vivo genome editing of PCSK9. Mol Ther. 6, 2019-2029 (2021).

Gorsuch, C. et al. A Gene Editing Approach to Eliminate Hepatitis B Virus Using ARCUS Meganucleases. American Society of Gene and Cell Therapy 2020 Annual Meeting.

Li, H. et al. Engineering a Self-inactivating Adeno-associated Virus (AAV) Vector for ARCUS Nuclease Delivery. American Society of Gene and Cell Therapy 2020 Annual Meeting.

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