Preclinical development of autologous hematopoietic stem cell-based gene therapy for immune deficiencies: a journey from mouse cage to bed side

HIGHLIGHTS

  • who: Laura Garcia-Perez and colleagues from the Department of Immunohematology and Blood Transfusion, Leiden University Medical Center have published the research: Preclinical Development of Autologous Hematopoietic Stem Cell-Based Gene Therapy for Immune Deficiencies: A Journey from Mouse Cage to Bed Side, in the Journal: Pharmaceutics 2020, 12, 549 of /2020/
  • what: The aim is to provide guidance in the structure and the required data to start a clinical trial application, focusing on efficacy (in_vitro and in_vivo) and safety. This model has a big impact on assessing gene therapy potential in pre-clinical studies . . .

     

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