Preclinical Development of Autologous Hematopoietic Stem Cell-Based Gene Therapy for Immune Deficiencies: A Journey from Mouse Cage to Bed Side

Recent clinical trials using patient’s own corrected hematopoietic stem cells (HSCs), such as for primary immunodeficiencies (Adenosine deaminase (ADA) deficiency, X-linked Severe Combined Immunodeficiency (SCID), X-linked chronic granulomatous disease (CGD), Wiskott–Aldrich Syndrome (WAS)), have yi...

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Detalles Bibliográficos
Autores principales: Garcia-Perez, Laura, Ordas, Anita, Canté-Barrett, Kirsten, Meij, Pauline, Pike-Overzet, Karin, Lankester, Arjan, Staal, Frank J. T.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2020
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7357087/
https://www.ncbi.nlm.nih.gov/pubmed/32545727
http://dx.doi.org/10.3390/pharmaceutics12060549

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7357087/
https://www.ncbi.nlm.nih.gov/pubmed/32545727
http://dx.doi.org/10.3390/pharmaceutics12060549

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