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Genome Editing

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MPS I Trial in Oakland (SB-318)

Terminated
  • MPS I
  • SB-318
  • Oakland, California
    UCSF Benioff Children's Hospital Oakland
Jan 6, 2023

Mucopolysaccharidosis II, MPS II Trial in United States (SB-913)

Terminated
  • Mucopolysaccharidosis II
  • MPS II
  • SB-913
  • Oakland, California
  • +4 more
Oct 21, 2022

Hemophilia B Trial in Washington (SB-FIX)

Terminated
  • Hemophilia B
  • SB-FIX
  • Washington, District of Columbia
    Georgetown University Medical Center
Mar 8, 2022

(LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX

Active, not recruiting
  • Hemophilia B
  • +2 more
  • SB-318
  • +2 more
  • Oakland, California
  • +4 more
Jan 30, 2023

Natural History of Autosomal Dominant Hearing Loss

Recruiting
  • Hearing Loss
    • Bethesda, Maryland
      National Institutes of Health Clinical Center
    Aug 12, 2022

    Viral Keratitis, Blindness Eye, Herpes Simplex Virus Infection Trial in Shanghai (BD111 Adult single group Dose)

    Completed
    • Viral Keratitis
    • +3 more
    • BD111 Adult single group Dose
    • Shanghai, Shanghai, China
      Eye & Ent Hospital of Fudan University
    Aug 20, 2022

    Relapsed/Refractory Acute Myeloid Leukemia (AML) Trial in Dresden, Heidelberg (Donor-derived CD34+ HSC with CRISPR/Cas9-mediated

    Not yet recruiting
    • Relapsed/Refractory Acute Myeloid Leukemia (AML)
    • Donor-derived CD34+ HSC with CRISPR/Cas9-mediated CD33 deletion
    • Gemtuzumab Ozogamicin
    • Dresden, Germany
    • +1 more
    Dec 20, 2022

    Transfusion Dependent Beta-thalassemia Trial in United States (ST-400 Investigational product)

    Active, not recruiting
    • Transfusion Dependent Beta-thalassemia
    • ST-400 Investigational product
    • Los Angeles, California
    • +5 more
    Jul 6, 2022

    Drug Sensitive Neoadjuvant Chemotherapy Based on Organoid Versus

    Not yet recruiting
    • Advanced Gastric Carcinoma
    • PDO group
    • Traditional group
    • Shanghai, Sahnghai, China
      Shanghai Ruijin Hospttal
    Apr 22, 2022

    Examining Knowledge, Attitudes, and Beliefs of Sickle Cell

    Completed
    • Sickle Cell Disease
      • Bethesda, Maryland
        National Human Genome Research Institute (NHGRI)
      Feb 5, 2021

      l Gene Editing Therapy for Cardiovascular Disease

      Not yet recruiting
      • Atherosclerotic Cardiovascular Disease
      • +2 more
        • Auckland, New Zealand
        • +2 more
        Oct 31, 2023

        Acute Lymphoblastic Leukemia, Chronic Lymphocytic Leukemia, Non Hodgkin Lymphoma Trial (PACE CART19)

        Withdrawn
        • Acute Lymphoblastic Leukemia
        • +2 more
        • PACE CART19
        • (no location specified)
        Mar 9, 2022

        Congenital Hearing Loss Trial in Shanghai (HG205)

        Not yet recruiting
        • Congenital Hearing Loss
        • HG205
        • Shanghai, China
          Eye & ENT Hospital of Fudan University
        Aug 29, 2023

        ImplementingNational Biobank of PD With WGS and Functional

        Not yet recruiting
        • Parkinson Disease
        • REM Sleep Behavior Disorder
        • whole genome sequencing
        • Pozzilli, Italy
          IRCCS Neuromed
        Feb 13, 2023

        Gene Editing as a Therapeutic Approach for Rett Syndrome

        Recruiting
        • Rett Syndrome
        • Gene editing in vitro
        • Siena, Italy
          University of Siena
        Feb 13, 2023

        Neurodegenerative Disease, Hereditary, Mitochondrial Diseases, Optic Atrophy Trial in Buffalo (Mutation analysis)

        Recruiting
        • Neurodegenerative Disease, Hereditary
        • +2 more
        • Mutation analysis
        • Buffalo, New York
          UBMD Pediatrics
        Mar 3, 2022

        Duchenne Muscular Dystrophy Trial in Worcester (CRD-TMH-001)

        Enrolling by invitation
        • Duchenne Muscular Dystrophy
        • Worcester, Massachusetts
          UMass Chan Medical School
        Aug 22, 2022

        NSCLC, Metastatic Non Small Cell Lung Cancer, Stage IV NSCLC Trial in Duarte, Minneapolis (Fludarabine, Cyclophosphamide, CISH

        Not yet recruiting
        • Carcinoma, Non-Small-Cell Lung
        • +5 more
        • Duarte, California
        • +1 more
        Dec 7, 2022

        Phenylketonurias, PAH Deficiency, Phenylketonuria Trial in United States (HMI-103)

        Recruiting
        • Phenylketonurias
        • +2 more
        • Orange, California
        • +5 more
        Oct 26, 2022

        Intraoperative Blood Pressure Changes and Whole Exome Genome

        Not yet recruiting
        • the Association Between the Arterial Stiffness, Intraoperative Blood Pressure Changes and Genetic Effect
        • this study did not involve interventions; it only observational study
        • (no location specified)
        Feb 7, 2023

        Disorder of Sex Development, 46,XY Trial in Montpellier (Identify structural variants by Optical Genome Mapping of DNA extracted

        Not yet recruiting
        • Disorder of Sex Development, 46,XY
        • Identify structural variants by Optical Genome Mapping of DNA extracted from blood leukocytes
        • Montpellier, France
          University Hospital Montpellier
        May 11, 2023

        Neovascular Age-related Macular Degeneration(nAMD) Trial in Shanghai, Tianjin (HG202)

        Not yet recruiting
        • Neovascular Age-related Macular Degeneration(nAMD)
        • HG202
        • Shanghai, Shanghai, China
        • +1 more
        Sep 4, 2023

        Fertility Issues Trial in New York, Portland, Kirkland (Embryo genome sequencing, Parents genome sequencing)

        Not yet recruiting
        • Fertility Issues
        • Embryo genome sequencing
        • Parents genome sequencing
        • New York, New York
        • +2 more
        Feb 13, 2023

        B Acute Lymphoblastic Leukemia Trial in London (PBLTT52CAR19)

        Recruiting
        • B Acute Lymphoblastic Leukemia
        • London, United Kingdom
          Great Ormond Street Hospital
        Dec 15, 2021

        Study of the Role of Genetic Modifiers in Hemoglobinopathies

        Recruiting
        • Sickle Cell Disease
        • +3 more
        • GWAS
        • Boston, Massachusetts
        • +24 more
        Apr 4, 2023