Treatment of SCID Due to ADA Deficiency With Autologous Transplantation of Cord Blood or Hematopoietic CD 34+ Cells After Addition of a Normal Human ADA cDNA by the EFS-ADA Lentiviral Vector

计划入组 1 人 · 首次发布于 2013 年 12 月 30 日

Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene

计划入组 46 人 · 首次发布于 2013 年 5 月 13 日

Gene Therapy for Adenosine Deaminase Severe Combined Immune Deficiency Using Peripheral Blood and EFS ADA Vector

计划入组 20 人 · 首次发布于 2022 年 6 月 27 日

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

lentiviral vector gene CRISPR/Cas9 therapy

ADA lentiviral vector-transduced hematopoietic stem cell gene therapy(Fondazione Telethon)

Ada Health GmbH

Germany

ETABLISSEMENT FRANCAIS DU SANG

France

NATIONAL AYURVEDA RESEARCH INSTITUTE FOR VECTOR BORNE DISEASES

India

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

Researchers Enhance Lentiviral Vector Potency for Liver Gene Therapy

Genenta Science and Anemocyte Expand Partnership to Commercialize Lentiviral Vector Plasmid DNA Technology Platform