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Journal Articles

CRISPR/Cas9 Genome-Edited Autologous Hematopoietic Stem Cell Transplantation Therapies for Sickle Cell Disease

Yi Liu, Vionnie W. Yu, Qi Zhang, Madhura Panditrao, Yi Yang, Alberto Del Rio-Espinola, Daher Ibrahim Aibo, Azeddine Elhajouji, Konrad Mueller, Karin Abitorabi, Daniel O'Connell, Bo Han, Nishit Patel, Carsten Russ, Craig Mickanin, Timothy K. MacLachlan, Susan C. Stevenson

Blood (2022) 140 (Supplement 1): 12982–12983.

https://doi.org/10.1182/blood-2022-159892

Published: 2022

Journal Articles

Treatment of Individuals with Severe Sickle Cell Disease with OTQ923, an Autologous, Ex Vivo , CRISPR/Cas9-Edited, CD34+ Hematopoietic Stem and Progenitor Cell Product, Leads to Durable Engraftment and Fetal Hemoglobin Induction

Akshay Sharma, Jaap Jan Boelens, Maria I. Cancio, Jane Hankins, Prafulla A Bhad, Andrew Lewandowski, Xiaojun Zhao, Shripad Chitnis, Radhika Peddinti, Yan Zheng, Neena Kapoor, Fabio Ciceri, Jianping Yuan, Vionnie W. Yu, Susan C. Stevenson, Serena De Vita, James LaBelle

Blood (2022) 140 (Supplement 1): 1906–1908.

https://doi.org/10.1182/blood-2022-166254

Published: 2022