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

Chronic granulomatous disease (CGD) is an inherited blood disorder of phagocytic cells that renders patients susceptible to infections and inflammation. A recent clinical trial of lentiviral gene therapy for the most frequent form of CGD, X-linked, has demonstrated stable correction over time, with...

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Detalles Bibliográficos
Autores principales: 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Mary Ann Liebert, Inc., publishers 2021
Materias:
Clinical Developments
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8575060/
https://www.ncbi.nlm.nih.gov/pubmed/33740872
http://dx.doi.org/10.1089/hum.2020.276

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8575060/
https://www.ncbi.nlm.nih.gov/pubmed/33740872
http://dx.doi.org/10.1089/hum.2020.276

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