An improved medium formulation for efficient ex vivo gene editing, expansion and engraftment of hematopoietic stem and progenitor cells

por Rai, Rajeev, Naseem, Asma, Vetharoy, Winston, Steinberg, Zohar, Thrasher, Adrian J., Santilli, Giorgia, Cavazza, Alessia
Publicado 2023

Targeted gene correction of human hematopoietic stem cells for the treatment of Wiskott - Aldrich Syndrome

por Rai, Rajeev, Romito, Marianna, Rivers, Elizabeth, Turchiano, Giandomenico, Blattner, Georges, Vetharoy, Winston, Ladon, Dariusz, Andrieux, Geoffroy, Zhang, Fang, Zinicola, Marta, Leon-Rico, Diego, Santilli, Giorgia, Thrasher, Adrian J., Cavazza, Alessia
Publicado 2020

Preclinical Optimization and Safety Studies of a New Lentiviral Gene Therapy for p47(phox)-Deficient Chronic Granulomatous Disease

por Schejtman, Andrea, Vetharoy, Winston, Choi, Uimook, Rivat, Christine, Theobald, Narda, Piras, Giuseppa, Leon-Rico, Diego, Buckland, Karen, Armenteros-Monterroso, Elena, Benedetti, Sara, Ashworth, Michael T., Rothe, Michael, Schambach, Axel, Gaspar, Hubert Bobby, Kang, Elizabeth M., Malech, Harry L., Thrasher, Adrian J., Santilli, Giorgia
Publicado 2021