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Gene Transfer

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Long-Term Follow-Up of Recipient of Gene Transfer Research

Recruiting
  • Retroviridae Infections
  • Cancer
  • Blood Tests
  • Questionnaires
  • Houston, Texas
    University of Texas MD Anderson Cancer Center
Aug 22, 2022

Parkinson Disease Trial in United States (AAV-GAD)

Enrolling by invitation
  • Parkinson Disease
  • AAV-GAD
  • West Bloomfield, Michigan
  • +4 more
Jun 7, 2023

Parkinson Disease Trial in West Bloomfield, Philadelphia, Morgantown (AAV-GAD, Sham Surgery)

Recruiting
  • Parkinson Disease
  • AAV-GAD
  • Sham Surgery
  • West Bloomfield, Michigan
  • +2 more
Jan 2, 2023

Spinal Muscular Atrophy 1 Trial in Columbus (AVXS-101)

Completed
Has Results
  • Spinal Muscular Atrophy 1
  • AVXS-101
  • Columbus, Ohio
    Nationwide Children's Hospital
Aug 30, 2022

Krabbe Disease Trial in Ann Arbor (FBX-101)

Recruiting
  • Krabbe Disease
  • FBX-101
  • Ann Arbor, Michigan
    University of Michigan Hospitals - Michigan Medicine
Aug 24, 2022

Subjects Previously Enrolled in Poxviral Vector Gene Transfer

Recruiting
  • Prostate Cancer
  • +4 more
    • Bethesda, Maryland
      National Institutes of Health Clinical Center
    Aug 19, 2022

    Limb Girdle Muscular Dystrophy Trial in Columbus (SRP-9003)

    Enrolling by invitation
    • Limb Girdle Muscular Dystrophy
    • SRP-9003
    • Columbus, Ohio
      Nationwide Children's Hospital
    May 17, 2023

    Limb Girdle Muscular Dystrophy Trial in Columbus (SRP-6004)

    Enrolling by invitation
    • Limb Girdle Muscular Dystrophy
    • SRP-6004
    • Columbus, Ohio
      Nationwide Children's Hospital
    Jun 7, 2023

    Hemophilia B Trial in United Kingdom, United States (Gene Transfer, scAAV2/8-LP1-hFIXco)

    Active, not recruiting
    • Hemophilia B
    • Stanford, California
    • +7 more
    Nov 17, 2022

    Severe Combined Immunodeficiency Disease, X-linked Trial in San Francisco, Memphis, Seattle (genetic, drug, device)

    Recruiting
    • Severe Combined Immunodeficiency Disease, X-linked
    • CL20-i4-EF1α-hγc-OPT
    • +2 more
    • San Francisco, California
    • +2 more
    Jan 9, 2023

    MPS IIIA, Sanfilippo Syndrome, Sanfilippo A Trial in Pittsburgh, North Adelaide, Santiago De Compostela (ABO-102)

    Terminated
    • MPS IIIA
    • +3 more
    • Pittsburgh, Pennsylvania
    • +2 more
    Jan 11, 2023

    MPS IIIA, Sanfilippo Syndrome, Sanfilippo A Trial in Australia, Spain, United States (ABO-102)

    Active, not recruiting
    • MPS IIIA
    • +3 more
    • ABO-102
    • Columbus, Ohio
    • +4 more
    Nov 8, 2022

    Lysosomal Diseases, Gangliosidosis, GM1 Trial run by the NHGRI (biological, drug, diagnostic test, other, procedure, behavioral)

    Recruiting
    • Lysosomal Diseases
    • +2 more
    • AAV9-GLB1
    • +20 more
    • Bethesda, Maryland
      National Institutes of Health Clinical Center
    Jan 19, 2023

    X-linked Severe Combined Immunodeficiency (XSCID) Trial run by the National Institute of Allergy and Infectious Diseases (NIAID)

    Suspended
    • X-linked Severe Combined Immunodeficiency (XSCID)
    • Bethesda, Maryland
      National Institutes of Health Clinical Center, 9000 Rockville Pi
    Aug 24, 2022

    Duchenne Muscular Dystrophy Trial in Columbus (rAAVrh74.MCK.GALGT2)

    Active, not recruiting
    Has Results
    • Duchenne Muscular Dystrophy
    • rAAVrh74.MCK.GALGT2
    • Columbus, Ohio
      Nationwide Children's Hospital
    Jul 26, 2022

    Multiple System Atrophy Trial in Irvine, Columbus (AAV2-GDNF gene therapy, Sham (Placebo) Surgery)

    Recruiting
    • Multiple System Atrophy
    • AAV2-GDNF gene therapy
    • Sham (Placebo) Surgery
    • Irvine, California
    • +1 more
    Jan 18, 2023

    Severe Combined Immunodeficiency, X Linked, Gene Therapy Trial in United Kingdom, United States (autologous CD34+ cell

    Recruiting
    • Severe Combined Immunodeficiency, X Linked
    • Gene Therapy
    • autologous CD34+ cell transduced with G2SCID vector
    • Los Angeles, California
    • +4 more
    Jul 19, 2022

    Hereditary Pulmonary Alveolar Proteinosis Trial (Gene-Corrected Macrophages administered by bronchoscopic instillation)

    Not yet recruiting
    • Hereditary Pulmonary Alveolar Proteinosis
    • Gene-Corrected Macrophages administered by bronchoscopic instillation
    • (no location specified)
    Mar 8, 2023

    Mucopolysaccharidosis Type 3 B Trial in Worldwide (rAAV9.CMV.hNAGLU)

    Terminated
    • Mucopolysaccharidosis Type 3 B
    • rAAV9.CMV.hNAGLU
    • Columbus, Ohio
    • +3 more
    Apr 29, 2022

    Sickle Cell Disease Trial in Los Angeles, Boston (single infusion of autologous bone marrow derived CD34+ HSC cells transduced

    Active, not recruiting
    • Sickle Cell Disease
    • single infusion of autologous bone marrow derived CD34+ HSC cells transduced with the lentiviral vector containing a short-hairpin RNA targeting BCL11a
    • Los Angeles, California
    • +1 more
    Feb 8, 2022

    Sickle Cell Disease Trial in Boston (Autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA

    Recruiting
    • Sickle Cell Disease
    • Autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA targeting BCL11a
    • Los Angeles, California
    • +8 more
    Jan 10, 2023

    CLN3, Batten Disease Trial in Columbus (AT-GTX-502)

    Active, not recruiting
    • CLN3
    • Batten Disease
    • AT-GTX-502
    • Columbus, Ohio
      Nationwide Children's Hospital
    Jul 18, 2022

    Hemophilia A Trial in Milwaukee (Auto CD34+PBSC, transduced with a lentiviral vector encoding the B domain deleted from of human

    Recruiting
    • Hemophilia A
    • Auto CD34+PBSC, transduced with a lentiviral vector encoding the B domain deleted from of human coagulation factor VIII
    • Milwaukee, Wisconsin
      Froedtert Hospital and the Medical College of Wisconsin
    Dec 21, 2022

    CLN6 Batten Disease Subjects Following Gene Transfer

    Active, not recruiting
    • CLN6
    • Batten Disease
    • AT-GTX-501
    • Columbus, Ohio
      Nationwide Children's Hosptial
    Dec 17, 2021

    Krabbe Disease Trial in Ann Arbor (FBX-101)

    Recruiting
    • Krabbe Disease
    • FBX-101
    • Ann Arbor, Michigan
      University of Michigan Hospitals - Michigan Medicine
    Feb 13, 2023