BioengineeringbioRxiv
Therapeutic strategy for spinal muscular atrophy by combining gene supplementation and genome editing
Fumiyuki Hatanaka, Keiichiro Suzuki, Kensaku Shojima…·Juan Carlos Izpisua Belmonte
The paper combines gene supplementation and genome editing to treat spinal muscular atrophy in mice, showing long-term therapeutic benefits.
DOI 10.1038/s41467-024-50095-5