3 studies found for:    23845948 [PUBMED-IDS]
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Rank Status Study
1 Recruiting Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy
Conditions: Metachromatic Leukodystrophy;   Adrenoleukodystrophy
Intervention: Genetic: transduced CD34+ hematopoietic stem cell
2 Recruiting Gene Therapy for Transfusion Dependent Beta-thalassemia
Condition: Beta-Thalassemia
Intervention: Genetic: Autologous hematopoietic stem cells genetically modified with GLOBE lentiviral vector encoding for the human beta-globin gene
3 Active, not recruiting Gene Therapy for Metachromatic Leukodystrophy
Condition: Metachromatic Leukodystrophy
Intervention: Genetic: Autologous CD34+ stem cells transduced with ARSA encoding lentiviral vector

Indicates status has not been verified in more than two years