Development and IND-enabling Studies of a Novel Cas9 Genome-Edited Autologous CD34+ Cell Therapy to Induce Fetal Hemoglobin for Sickle Cell Disease
Varun Katta,Kiera O'Keefe,Yichao Li,Thiyagaraj Mayuranathan,Cicera R. Lazzarotto, Rachael K. Wood,Rachel M. Levine,Alicia Powers,Kalin Mayberry, Garret Manquen,Yu Yao,Jingjing Zhang,Yoonjeong Jang,Nikitha Nimmagadda,Erin A. Dempsey,GaHyun Lee,Naoya Uchida,Yong Cheng,Frank Fazio, Tim Lockey, Mike Meagher,Akshay Sharma,John F. Tisdale,Sheng Zhou,Jonathan S. Yen,Mitchell J. Weiss,Shengdar Q. Tsai Molecular Therapy（2024）
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