#cystinosis — Thursday, February 19, 2026 (1 items)
| 0 | ℹ |
Hematopoietic Stem-Cell Gene Therapy for Cystinosis.
(nejm.org)
|
New England Journal of Medicine 2026-02-19 | #gene therapy #hematopoietic stem cells #lysosomal-storage-disorder #lentiviral-vectors #cystinosis |