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Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease |
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Titel: |
Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease |
Auteur: |
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. |
Verschenen in: |
Molecular therapy |
Paginering: |
Jaargang 32 () nr. 10 pagina's 3433-3452 |
Jaar: |
2024 |
Inhoud: |
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Uitgever: |
The Authors |
Bronbestand: |
Elektronische Wetenschappelijke Tijdschriften |
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