Tag: cystinosis

Oophorectomy associated with reduced cancer risk and all-cause mortality for BRCA1/2 patients

Ex vivo gene therapy for cystinosis had acceptable safety and improved disease biomarkers

byZhenyu Liand1 others

March 3, 2026

1. In this phase 1–2 open-label study, autologous lentiviral hematopoietic stem-cell gene therapy for cystinosis was associated with sustained engraftment ...

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