Molecular and Clinical Studies of Primary Immunodeficiency Diseases

计划入组 266 人 · 首次发布于 2000 年 10 月 2 日

MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID

计划入组 10 人 · 首次发布于 2008 年 11 月 20 日

Gene Therapy for ADA-SCID

计划入组 8 人 · 首次发布于 2008 年 1 月 24 日

ADA lentiviral vector-transduced stem cell gene therapy (ADA-SCID)

VM-105

ADA-308

Ada Health GmbH

Germany

American Dental Association

United States

ILLINOIS INSTITUTE OF TECHNOLOGY

United States

UCLA Gene Therapy Achieves 100% Cure Rate for Bubble Baby Disease in Clinical Trials

Gene Therapy Achieves 95% Success Rate in Treating ADA-SCID 'Bubble Boy' Disease

AGC Biologics Partners with Rarity PBC to Advance First Commercial Gene Therapy for ADA-SCID "Bubble Baby Disease"