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

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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Bibliographic Details
Main Authors: Laura Garcia-Perez, Anita Ordas, Kirsten Canté-Barrett, Pauline Meij, Karin Pike-Overzet, Arjan Lankester, Frank J. T. Staal
Format: Article
Language:English
Published: MDPI AG 2020-06-01
Series:Pharmaceutics
Subjects:
gene therapy
immunodeficiency
HSC
vector design
animal model
efficacy
Online Access:https://www.mdpi.com/1999-4923/12/6/549

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https://www.mdpi.com/1999-4923/12/6/549

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