Scalable GMP-compliant gene correction of CD4+ T cells with IDLV template functionally validated in vitro and in vivo

Abstract excerpt

Hyper-IgM1 is a rare X-linked combined immunodeficiency caused by mutations in the CD40 ligand ( CD40LG ) gene with a median survival of 25 years, potentially treatable with in situ CD4+ T cell gene editing with Cas9 and a one-size-fits-most corrective donor template. Here, starting from our research-grade editing protocol, we pursued the development of a good manufacturing practice (GMP)-compliant, scalable...

Topics

Share this publication in a Topic to start or enrich a Post.

• Share to CAR-T cell therapy research
• Share to CRISPR and Genetic Engineering
• Share to Virus-based gene therapy research