In Vivo Outcome of Homology-Directed Repair at the HBB Gene in HSC Using Alternative Donor Template Delivery Methods

by Sowmya Pattabhi, Samantha N. Lotti, Mason P. Berger, Swati Singh, Christopher T. Lux, Kyle Jacoby, Calvin Lee, Olivier Negre, Andrew M. Scharenberg, David J. Rawlings
Published 2019-09-01

pEVL: A Linear Plasmid for Generating mRNA IVT Templates With Extended Encoded Poly(A) Sequences

by Alexandra E Grier, Stephen Burleigh, Jaya Sahni, Courtnee A Clough, Victoire Cardot, Dongwook C Choe, Michelle C Krutein, David J Rawlings, Michael C Jensen, Andrew M Scharenberg, Kyle Jacoby
Published 2016-01-01

TALEN-Mediated Gene Editing of HBG in Human Hematopoietic Stem Cells Leads to Therapeutic Fetal Hemoglobin Induction

by Christopher T. Lux, Sowmya Pattabhi, Mason Berger, Cynthia Nourigat, David A. Flowers, Olivier Negre, Olivier Humbert, Julia G. Yang, Calvin Lee, Kyle Jacoby, Irwin Bernstein, Hans-Peter Kiem, Andrew Scharenberg, David J. Rawlings
Published 2019-03-01