REP1 Gene Replacement Therapy for Choroideremia

计划入组 30 人 · 首次发布于 2015 年 4 月 3 日

Gene Replacement Therapy for Treatment of Paediatric Patients With CTNNB1 Neurodevelopmental Syndrome

计划入组 12 人 · 首次发布于 2025 年 12 月 8 日

Safety Study of rAAV2/8-hCYP4V2 in Patients With Bietti's Crystalline Dystrophy (BCD)

计划入组 12 人 · 首次发布于 2021 年 1 月 25 日

scAAV2/MFSD8 gene replacement therapy

AAV9-based gene replacement therapy (CLN7 disease)

AAV9-based gene replacement therapy (University of Texas Southwestern Medical Center)

CELL AND GENE THERAPY CATAPULT

United Kingdom

COLORADO JOINT REPLACEMENT

United States

JIUYUAN GENE

Genezen and Raiden Science Foundation Partner to Advance First AAV9 Gene Replacement Therapy for UBA5 Disorder Toward Clinical Trial

UC Davis Health Premieres Documentary on Duchenne Muscular Dystrophy Gene Therapy Trial

Nuevocor Receives FDA IND Clearance for Gene Therapy Targeting Rare Genetic Heart Disease