Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease
Katta, Varun, O’Keefe, Kiera, Li, Yichao, Mayuranathan, Thiyagaraj, Lazzarotto, Cicera R., Wood, Rachael K., Levine, Rachel M., Powers, Alicia, Mayberry, Kalin, Manquen, Garret, Yao, Yu, Zhang, Jingjing, Jang, Yoonjeong, Nimmagadda, Nikitha, Dempsey, Erin A., Lee, GaHyun, Uchida, Naoya, Cheng, Yong, Fazio, Frank, Lockey, Tim, Meagher, Mike, Sharma, Akshay, Tisdale, John F., Zhou, Sheng, Yen, Jonathan S., Weiss, Mitchell J., Tsai, Shengdar Q.
Published in Molecular therapy (02.10.2024)
Published in Molecular therapy (02.10.2024)
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