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

• Egile Nagusiak: Laura Garcia-Perez (Egilea), Anita Ordas (Egilea), Kirsten Canté-Barrett (Egilea), Pauline Meij (Egilea), Karin Pike-Overzet (Egilea), Arjan Lankester (Egilea), Frank J. T. Staal (Egilea)
• Formatua: Liburua
• Argitaratua: MDPI AG, 2020-06-01T00:00:00Z.
• Gaiak: article
• Sarrera elektronikoa: Connect to this object online.

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)), ha...