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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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Detaylı Bibliyografya
Yayımlandı:Pharmaceutics
Asıl Yazarlar: Garcia-Perez, Laura, Ordas, Anita, Canté-Barrett, Kirsten, Meij, Pauline, Pike-Overzet, Karin, Lankester, Arjan, Staal, Frank J. T.
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: MDPI 2020
Konular:
Online Erişim:https://ncbi.nlm.nih.gov/pmc/articles/PMC7357087/
https://ncbi.nlm.nih.gov/pubmed/32545727
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/pharmaceutics12060549
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