239. Preclinical Studies for the First Hematopoietic Stem...

Molecular Therapy 2015 / 05 Vol. 23

239. Preclinical Studies for the First Hematopoietic Stem Cell (HSC) Gene Editing Trial: Phase 1 Study of Beta-Thalassemia With Autologous Transplantation of Zinc Finger Nuclease-Treated HSC To Upregulate Fetal Hemoglobin

Ando, Dale, Walters, Mark C., Chang, Kai-Hsin, Reik, Andreas, Urnov, Fyodor D., Lee, Gary, Spratt, Kaye S., Rebar, Edward J., Gregory, Philip D., Tang, Winson, Papayannopoulou, Thalia, Stamatoyannopou
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Volume:
23
Language:
english
Journal:
Molecular Therapy
DOI:
10.1016/s1525-0016(16)33844-8
Date:
May, 2015
File:
PDF, 290 KB
english, 2015
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