Gene therapy of RAG-2−/− mice: sustained correction of the immunodeficiency

Abstract excerpt

Patients with mutations of either RAG-1 or RAG-2 genes suffer from severe combined immunodeficiency (SCID) characterized by the lack of T and B lymphocytes. The only curative treatment today consists of hematopoietic stem cell (HSC) transplantation, which is only partially successful in the absence of an HLA genoidentical donor, thus justifying research to find an alternative therapeutic approach. To this end,...

Topics

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

• Share to CAR-T cell therapy research
• Share to Immunodeficiency and Autoimmune Disorders
• Share to Virus-based gene therapy research