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gene correction

Showing 1 - 25 of 7,486
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Sickle Cell Disease Trial in Birmingham, Palo Alto, Saint Louis (GPH101 Drug Product)

Active, not recruiting
  • Sickle Cell Disease
  • GPH101 Drug Product
  • Birmingham, Alabama
  • +2 more
Jan 5, 2023

Correction of Nonsense Mutations in Cystic Fibrosis

Recruiting
  • Cystic Fibrosis
  • smear of nasal fossae
  • Amiens, France
  • +7 more
Nov 17, 2020

Gene Editing as a Therapeutic Approach for Rett Syndrome

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

Genetic Regulators of Bone Health That Are Unique to Vertebral

Not yet recruiting
  • Age-Related Osteoporosis
  • Sample collection for gene expression
  • Aurora, Colorado
    Univeristy of Colorado Denver
Jul 17, 2023

Gene Expression Trial in Miami (Sculptra, Radiesse Plus)

Active, not recruiting
  • Gene Expression
  • Sculptra
  • Radiesse Plus
  • Miami, Florida
    Miami Dermatology & Laser Institute
Jan 5, 2023

MPS-IH (Hurler Syndrome) Trial in Worldwide (Experimental: OTL-203, Active Comparator: Allo-HSCT)

Not yet recruiting
  • MPS-IH (Hurler Syndrome)
  • Experimental: OTL-203
  • Active Comparator: Allo-HSCT
  • Oakland, California
  • +6 more
Nov 27, 2023

Lysosomal Storage Disease, Metachromatic Leukodystrophy Trial in Milan (OTL-200 Gene Therapy)

Active, not recruiting
  • Lysosomal Storage Disease
  • Metachromatic Leukodystrophy
  • OTL-200 Gene Therapy
  • Milan, Italy
    Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR
Nov 4, 2022

Pyruvate Kinase Deficiency Trial in Stanford, Madrid (RP-L301)

Recruiting
  • Pyruvate Kinase Deficiency
  • RP-L301
  • Stanford, California
  • +2 more
Sep 22, 2021

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

Fibroblast-Derived Cell Lines From Skin Biopsies of

Enrolling by invitation
  • Primary Immunodeficiency
  • +2 more
    • Bethesda, Maryland
      National Institutes of Health Clinical Center
    Jan 18, 2023

    Chronic Rhinosinusitis With Nasal Polyps Trial in Vancouver (Mepolizumab)

    Not yet recruiting
    • Chronic Rhinosinusitis With Nasal Polyps
    • Vancouver, British Columbia, Canada
      St. Paul's Sinus Centre
    Oct 24, 2023

    Spinal Cord Injuries Trial in Iowa City (Single-session electrically induced exercise, Electrically-induced exercise training)

    Completed
    Has Results
    • Spinal Cord Injuries
    • Single-session electrically induced exercise
    • Electrically-induced exercise training
    • Iowa City, Iowa
      University of Iowa
    Oct 6, 2022

    Thalassemia Trial (iHSCs treatment group)

    Unknown status
    • Thalassemia
    • iHSCs treatment group
    • (no location specified)
    Nov 1, 2018

    Colorectal Cancer, Anemia, Iron-deficiency Trial (Iron(III)isomaltoside)

    Not yet recruiting
    • Colorectal Cancer
    • +2 more
    • (no location specified)
    Jan 21, 2022

    Preterm, Nutritional Deficiency, Immune System and Related Disorders Trial in Liverpool (Arginine)

    Recruiting
    • Preterm
    • +2 more
    • Arginine
    • Liverpool, Merseyside, United Kingdom
      Liverpool Women's Hospital
    Mar 17, 2022

    Gastric, Colorectal Adenocarcinoma Trial in Shenyang (tislelizumab)

    Not yet recruiting
    • Gastric
    • Colorectal Adenocarcinoma
    • Shenyang, Liaoning, China
      Liaoning Cancer Hospital & Institute
    Nov 1, 2023

    Adenosine Deaminase Severe Combined Immune Deficiency Trial (A cryopreserved formulation of autologous mPB CD34+ hematopoietic

    Not yet recruiting
    • Adenosine Deaminase Severe Combined Immune Deficiency
    • A cryopreserved formulation of autologous mPB CD34+ hematopoietic stem and progenitor cells transduced ex vivo with the EFS-ADA lentiviral vector encoding the human ADA enzyme
    • (no location specified)
    Jun 20, 2022

    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

    Recurrent Plasma Cell Myeloma, Refractory Plasma Cell Myeloma Trial in Atlanta, Ann Arbor, Saint Louis (Dexamethasone, Ixazomib,

    Active, not recruiting
    • Recurrent Plasma Cell Myeloma
    • Refractory Plasma Cell Myeloma
    • Atlanta, Georgia
    • +3 more
    Jan 6, 2023

    Apheresis of Patients With Immunodeficiency

    Recruiting
    • LAD-1
    • +2 more
      • Bethesda, Maryland
        National Institutes of Health Clinical Center, 9000 Rockville Pi
      Aug 24, 2022

      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

      Peripheral Blood Hematopoietic Stem Cells, Mononuclear Cells and

      Recruiting
      • Granuloma
      • +4 more
        • Bethesda, Maryland
          National Institutes of Health Clinical Center
        Jan 25, 2023

        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

        Metachromatic Leukodystrophy, Adrenoleukodystrophy Trial in Shenzhen (transduced CD34+ hematopoietic stem cell)

        Recruiting
        • Metachromatic Leukodystrophy
        • Adrenoleukodystrophy
        • transduced CD34+ hematopoietic stem cell
        • Shenzhen, Guangdong, China
          Shenzhen Second People's Hospital, The First Affiliated Hospital
        May 25, 2022