Safety and Efficacy of CTX001 in Patients with Transfusion-Dependent β-Thalassemia (TDT) or Sickle Cell Disease (SCD): Early Results from the Climb THAL-111 and Climb SCD-121 Studies of Autologous CRISPR-Cas9-Modified CD34+ Hematopoietic Stem and Progenitor Cells (HSPCs)

Autor:	Yimeng Lu, Markus Y. Mapara, Niraj Shanbhag, Damiano Rondelli, Franco Locatelli, C. Fernández, Sandeep Soni, Donna A. Wall, Yael Bobruff, John K. Wu, Stephan A. Grupp, Sarah D. Mulcahey, Sujit Sheth, Haydar Frangoul, Antonis Kattamis, Maria Domenica Cappellini, Tony W. Ho, J Lekstrom-Himes, Mariane de Montalembert, Martin H. Steinberg, Selim Corbacioglu, Josu de la Fuente, Rupert Handgretinger, Michael J. Weinstein, Suzan Imren
Rok vydání:	2021
Předmět:	Transplantation business.industry Thalassemia Cell CD34 Cell Biology Hematology Disease medicine.disease Haematopoiesis medicine.anatomical_structure Immunology Molecular Medicine Immunology and Allergy Medicine CRISPR In patient Progenitor cell business
Zdroj:	Transplantation and Cellular Therapy. 27:S72-S73
ISSN:	2666-6367
DOI:	10.1016/s2666-6367(21)00101-9
Databáze:	OpenAIRE
Externí odkaz:	https://explore.openaire.eu/search/publication?articleId=doi_________::89e261f83fae91cabdc601c32eeadf0e https://doi.org/10.1016/s2666-6367(21)00101-9 Zobrazit plný text záznamu Full Text from ScienceDirect