Non-genotoxic conditioning facilitates hematopoietic stem cell gene therapy for hemophilia A using bioengineered factor VIII

Hematopoietic stem and progenitor cell (HSPC) lentiviral gene therapy is a promising strategy toward a lifelong cure for hemophilia A (HA). The primary risks associated with this approach center on the requirement for pre-transplantation conditioning necessary to make space for, and provide immune s...

ver descrição completa

Detalhes bibliográficos

Principais autores:	Athena L. Russell, Chengyu Prince, Taran S. Lundgren, Kristopher A. Knight, Gabriela Denning, Jordan S. Alexander, Jaquelyn T. Zoine, H. Trent Spencer, Shanmuganathan Chandrakasan, Christopher B. Doering
Formato:	Artigo
Idioma:	Inglês
Publicado em:	Elsevier 2021-06-01
coleção:	Molecular Therapy: Methods & Clinical Development
Assuntos:	non-genotoxic conditioning antibody conditioning immunotoxin antibody-drug conjugate hematopoietic stem cell transplantation ex vivo lentiviral vector gene therapy
Acesso em linha:	http://www.sciencedirect.com/science/article/pii/S2329050121000826
Tags:	Adicionar Tag Sem tags, seja o primeiro a adicionar uma tag!