Phase 1-2 study | Lentiviral gene therapy for artemis-deficient SCID

Lentiviral Gene Therapy for Artemis-Deficient SCID – New England Journal of Medicine (link to abstract – $ for full-text)

Commentary: Infant gene therapy is a breakthrough for Artemis-SCID patients – University of California – San Francisco

Commentary on Twitter

Severe combined immunodeficiency caused by mutations in the gene encoding a DNA-repair enzyme called Artemis is difficult to treat. This study involving 10 patients supports a gene-therapy approach. https://t.co/SCpZjOQtMW pic.twitter.com/VRYOje6UPV

— NEJM (@NEJM) December 22, 2022