Gene Therapy for Fanconi Anemia, Complementation Group a: Updated Results from Ongoing Global Clinical Studies of RP-L102

• Published in: Blood Vol. 136; no. Supplement 1; p. 14
• Main Authors: Czechowicz, Agnieszka, Agarwal, Rajni, Sevilla, Julián, Río, Paula, Navarro, Susana, Beard, Brian C, Law, Kenneth M, Choi, Grace, Zeini, Miriam, Nicoletti, Eileen, Wagner, John E, Booth, Claire, Schwartz, Jonathan D, Bueren, Juan A, Roncarolo, Maria Grazia
• Format: Journal Article
• Language: English
• Published: Elsevier Inc 05.11.2020

Background: Fanconi anemia (FA) is a rare inherited disorder of defective cellular deoxyribonucleic acid (DNA) repair, associated with developmental abnormalities and characterized by progressive bone marrow failure (BMF) and a predisposition to hematologic malignancies and solid tumors. Approximate...