Gene-Edited CAR T Expands Rare Disease Horizons

Gene-Edited CAR T Expands Rare Disease Horizons

For more than a decade, chimeric antigen receptor T-cell (CAR T) therapy has transformed the treatment landscape for certain blood cancers. Now, researchers believe the next major leap might come from combining CAR T with powerful gene-editing technologies such as CRISPR (which stands for clustered regularly interspaced short palindromic repeats) and TALEN (which stands for transcription activator-like effector nucleases), opening new possibilit…

Clinical trial combines CRISPR gene editing with stem cell transplants to treat aggressive blood cancers

Researchers used CRISPR to remove the CD33 protein from donor stem cells, protecting healthy blood cells from therapies that target leukemia. In a 30-patient trial, the edited cells rebuilt blood production normally and allowed some patients to receive CD33-targeted maintenance therapy without prolonged severe blood-cell suppression. The approach could make CAR-T and other targeted treatments safer for AML and MDS, though larger studies and long…