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GENE THERAPY

Publisher:
Springer Nature
ISSN:
0969-7128
Category:
MEDICINE, RESEARCH & EXPERIMENTAL
Impact factor:
4.6

Feed status

3 parsed articles

Last update: Not fetched

Latest articles

First-in-human nuclease-free homologous recombination-dependent gene editing in pediatric patients with methylmalonic acidemia: results of a phase 1/2 study

2026-03-30

Jirair K. Bedoyan, Thomas Morgan, Angela Sun, Hong Li, Daniel Gruskin, Marie Payton, Frederic Chereau, Eugene Scott Swenson, Qun Lin, Mark A. Kay, Jerry Vockley

Gene Therapy, Published online: 30 March 2026; doi:10.1038/s41434-026-00609-1 First-in-human nuclease-free homologous recombination-dependent gene editing in pediatric patients with methylmalonic acidemia: results of a phase 1/2 study