Successful Preclinical Development of Gene Therapy for RAG1-SCID
Recomb partner, Sheba published an article on a Large Cohort of RAG1/2-Deficient SCID Patients
March 11, 2020ESGCT e-School
May 18, 2020
The latest publication by Garcia-Perez from the Staal group/LUMC, in collaboration with other RECOMB partners, reports about the successful preclinical development of gene therapy for RAG1-SCID. The article discusses efficacy outcome and safety data of the preclinical development phase, and emphasizes that the results substantiate the need for a clinical trial for RAG1-SCID which is the main objective of the RECOMB consortium. Read this great article here.
