Found 58 matching trials
Gene-Modified Stem Cell Therapy for Children With Autosomal Recessive Osteopetrosis (ARO) PHASE1 This is a non-randomized, one-arm, open label, single-center, phase I/II, p...
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Ospedale San Raffaele
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Study of Gene Therapy Using a Lentiviral Vector to Treat X-linked Chronic Granulomatous Disease PHASE1 Chronic Granulomatous Disease (CGD) is an inherited immunodeficiency disord...
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University of California, Los Angeles (UCLA)
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Safety Study of Zinc Finger Nuclease CCR5-modified Hematopoietic Stem/Progenitor Cells in HIV-1 Infected Patients PHASE1 The purpose of the study is to evaluate the safety and feasibility of admin...
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Active Not Recruiting
Location
City of Hope Medical Center
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Gene-Modified HIV-Protected Stem Cell Transplant in Treating Patients With HIV-Associated Lymphoma PHASE1 This clinical trial studies gene-modified, human immunodeficiency virus (HI...
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Fred Hutch/University of Washington Cancer Consortium
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Lentiviral Vector Gene Therapy - The Guard1 Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease PHASE1 This was a multinational, open-label study to assess the safety and efficac...
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University of California San Diego
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Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD) PHASE3 This is a single-dose, open-label study in pediatric participants with seve...
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Location
Levine Children's Hospital - Hematology
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Safety and Efficacy Evaluation of BRL-101 in Subjects With Transfusion-Dependent β-Thalassemia PHASE1 This is a non-randomized, open label, multi-site, single-dose, phase 1/2 st...
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Location
Nanfang Hospital, Southern Medical University
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Safety and Efficacy Study of KL-7SHRNA Injection Solution in the Treatment of AIDS Patients With Lymphoma NA This pilot clinical trial studies gene therapy in treating patients with HI...
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Affiliated hospital of Guangdong medical university
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Safety of Transplantation of CRISPR CCR5 Modified CD34+ Cells in HIV-infected Subjects With Hematological Malignances NA The investigators performed this study to evaluate the safety and feasibili...
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307 Hospital of PLA (Affiliated Hospital of Academy to Military Medical Sciences)
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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. PHASE1 The purpose of this study is to evaluate the Safety and Efficacy of DREAM01...
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Department of Biotherapy, Necker-Enfants Malades Hospital
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