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

• Principais autores: Laura Garcia-Perez (Autor), Anita Ordas (Autor), Kirsten Canté-Barrett (Autor), Pauline Meij (Autor), Karin Pike-Overzet (Autor), Arjan Lankester (Autor), Frank J. T. Staal (Autor)
• Formato: Livro
• Publicado em: MDPI AG, 2020-06-01T00:00:00Z.
• Assuntos: article
• Acesso em linha: 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...