Gene Transfer for Patients With Sickle Cell Disease

计划入组 7 人 · 首次发布于 2014 年 7 月 10 日

Efficacy Safety Study of Gene Therapy for Sickle Cell DiseaseSCD Using Autologous CD34+ Cells Transduced ex Vivo, Carrying a Corrected Globin Gene and a Silencing RNA.

计划入组 15 人 · 首次发布于 2026 年 2 月 25 日

Clinical Study of AAV1-gamma-sarcoglycan Gene Therapy for Limb Girdle Muscular Dystrophy Type 2C

计划入组 9 人 · 首次发布于 2011 年 4 月 29 日

AAV1-gamma-sarcoglycan vector(Genethon)

Interferon-gamma gene therapy(DNAtrix)

γ-globin gene therapy(CSL Behring LLC)

JIUYUAN GENE

Triple-Gene LLC

United States

MOMOTARO-GENE INC.

Japan

MiNA Therapeutics Reports Breakthrough Pre-Clinical Results for RNA-Based Sickle Cell Disease Treatment

Editas Medicine Advances EDIT-301 for Sickle Cell Disease and Beta Thalassemia, Prioritizes In Vivo Gene Editing

CRISPR-Based Casgevy Emerges as Preferred Gene Therapy for Beta-Thalassemia, Survey Shows