Evidence for Long-term Efficacy and Safety of Gene Therapy for Wiskott–Aldrich Syndrome in Preclinical Models

Wiskott–Aldrich Syndrome (WAS) is a life-threatening X-linked disease characterized by immunodeficiency, thrombocytopenia, autoimmunity, and malignancies. Gene therapy could represent a therapeutic option for patients lacking a suitable bone marrow (BM) donor. In this study, we analyzed the long-ter...

Полное описание

Сохранить в:
Библиографические подробности
Главные авторы: Marangoni, Francesco, Bosticardo, Marita, Charrier, Sabine, Draghici, Elena, Locci, Michela, Scaramuzza, Samantha, Panaroni, Cristina, Ponzoni, Maurilio, Sanvito, Francesca, Doglioni, Claudio, Liabeuf, Marie, Gjata, Bernard, Montus, Marie, Siminovitch, Katherine, Aiuti, Alessandro, Naldini, Luigi, Dupré, Loïc, Roncarolo, Maria Grazia, Galy, Anne, Villa, Anna
Формат: Artigo
Язык:Inglês
Опубликовано: Nature Publishing Group 2009
Предметы:
Original Articles
Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC2835187/
https://ncbi.nlm.nih.gov/pubmed/19259069
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2009.31
Метки: Добавить метку
Нет меток, Требуется 1-ая метка записи!

Схожие документы