Outcome following Gene Therapy in Patients with Severe Wiskott-Aldrich Syndrome

IMPORTANCE: Wiskott-Aldrich syndrome (WAS) is a rare primary immunodeficiency associated with severe microthrombocytopenia. Partially HLA-matched allogeneic hematopoietic stem cell (HSC) transplantation is associated with significant co-morbidity. OBJECTIVE: To assess the outcome and safety of autol...

ver descrição completa

Na minha lista:
Detalhes bibliográficos
Publicado no:JAMA
Main Authors: Abina, Salima Hacein-Bey, Gaspar, H. Bobby, Blondeau, Johanna, Caccavelli, Laure, Charrier, Sabine, Buckland, Karen, Picard, Capucine, Six, Emmanuelle, Himoudi, Nourredine, Gilmour, Kimberly, McNicol, Anne-Marie, Hara, Havinder, Xu-Bayford, Jinhua, Rivat, Christine, Touzot, Fabien, Mavilio, Fulvio, Lim, Annick, Treluyer, Jean-Marc, Héritier, Sébastien, Lefrere, Francois, Magalon, Jeremy, Pengue-Koyi, Isabelle, Honnet, Géraldine, Blanche, Stéphane, Sherman, Eric A., Male, Frances, Berry, Charles, Malani, Nirav, Bushman, Frederic D., Fischer, Alain, Thrasher, Adrian J., Galy, Anne, Cavazzana, Marina
Formato: Artigo
Idioma:Inglês
Publicado em: 2015
Assuntos:
Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4942841/
https://ncbi.nlm.nih.gov/pubmed/25898053
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1001/jama.2015.3253
Tags: Adicionar Tag
Sem tags, seja o primeiro a adicionar uma tag!

Registos relacionados