ESGCT e-SchoolMay 18, 2020
Commentary article on human T cell developmentSeptember 22, 2020
Garcia-Perez and colleagues from LUMC have recently published a paper discussing the journey of Preclinical Development of Autologous Hematopoietic Stem Cell-Based Gene Therapy for Immune Deficiencies. In this review, several different challenges are addressed regarding the development of gene therapy for immune deficiencies using their own experience with RAG1-SCID as an example (product development, the proof-of-concept (in vitro and in vivo efficacy, toxicology, and safety), and the final release steps to the clinic (scaling up, good manufacturing practice (GMP) procedures/protocols and regulatory hurdles). Read this great article here.