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Gene therapy

Showing 26 - 50 of >10,000
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Drug Resistant Epilepsy Trial (lentiviral gene therapy)

Not yet recruiting
  • Drug Resistant Epilepsy
  • lentiviral gene therapy
  • (no location specified)
May 17, 2022

Age-Related Macular Degeneration Trial in Beijing (KH631)

Not yet recruiting
  • Age-Related Macular Degeneration
  • Beijing, China
    Beijing Tongren Hospital, Capital Medical University
Jan 3, 2023

PDE6A-related Retinitis Pigmentosa in Preparation toGene Therapy

Enrolling by invitation
  • Retinitis Pigmentosa
    • Tübingen, Baden-Württemberg, Germany
      Institute for Ophthalmic Research, University Tübingen, Germany
    Oct 25, 2022

    Duchenne Muscular Dystrophy Trial in Little Rock (RGX-202)

    Recruiting
    • Duchenne Muscular Dystrophy
    • RGX-202
    • Little Rock, Arkansas
      Arkansas Children's Hospital
    Jan 12, 2023

    Trial

    Not yet recruiting
    • Leber Hereditary Optic Neuropathy (LHON)
    • NR082 Injection
    • Injection needle
    • (no location specified)
    Jan 30, 2023

    Muscular Atrophy, Spinal Trial (risdiplam)

    Not yet recruiting
    • Muscular Atrophy, Spinal
    • (no location specified)
    May 8, 2023

    Treatment of Symptoms of Grade 2 and Grade 3 Late Xerostomia From Salivary Gland Hypofunction Caused by Radiotherapy for Cancer

    Recruiting
    • Treatment of Symptoms of Grade 2 and Grade 3 Late Xerostomia From Salivary Gland Hypofunction Caused by Radiotherapy for Cancer of the Oropharynx
    • AAV2-hAQP1 Concentration 1
    • +2 more
    • Charlotte, North Carolina
      Atrium Health
    Jun 22, 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

    Knowledge, Beliefs, and Attitudes of Hemophilia B About Gene

    Recruiting
    • Gene Therapy
    • Hemophilia B
    • Interview
    • Memphis, Tennessee
      St. Jude Children's Research Hospital
    May 25, 2022

    Danon Disease Trial in La Jolla, Philadelphia (RP-A501)

    Recruiting
    • Danon Disease
    • RP-A501
    • La Jolla, California
    • +1 more
    Oct 17, 2023

    Fabry Disease Who Received Lentiviral Gene Therapy in Study

    Enrolling by invitation
    • Fabry Disease
    • Safety and Efficacy Assessments
    • Melbourne, Parkville VIC, Australia
    • +2 more
    Oct 5, 2022

    LGMD2C Trial (ATA-200)

    Not yet recruiting
    • LGMD2C
    • ATA-200
    • (no location specified)
    Aug 1, 2023

    Beta Thalassaemia Trial in Milan (Safety and Efficacy assessments)

    Active, not recruiting
    • Beta Thalassaemia
    • Safety and Efficacy assessments
    • Milan, Italy
      Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR
    Nov 22, 2022

    Malignant Glioma of Brain, Astrocytoma, Malignant Astrocytoma Trial in Detroit (Ad5-yCD/mutTKSR39rep-ADP adenovirus and

    Recruiting
    • Malignant Glioma of Brain
    • +8 more
    • Ad5-yCD/mutTKSR39rep-ADP adenovirus and fractionated stereotactic radiosurgery (fSRS)
    • Detroit, Michigan
      Henry Ford Health System
    Jan 9, 2023

    Spinal Muscular Atrophy Trial in Beijing, Shenyang, Suzhou (GC101)

    Recruiting
    • Spinal Muscular Atrophy
    • GC101
    • Beijing, China
    • +3 more
    Apr 22, 2023

    SMA II Trial in China (GC101)

    Not yet recruiting
    • SMA II
    • GC101
    • Beijing, China
    • +4 more
    Jun 15, 2023

    Phenylketonurias Trial in Bengbu (NGGT002)

    Recruiting
    • Phenylketonurias
    • NGGT002
    • Bengbu, Anhui, China
      The First Affiliated Hospital of Bengbu Medical College
    Sep 25, 2023

    Duchenne Muscular Dystrophy Trial in United States (EDG-5506 Dose 1, EDG-5506 Dose 2, Placebo)

    Not yet recruiting
    • Duchenne Muscular Dystrophy
    • EDG-5506 Dose 1
    • +2 more
    • Los Angeles, California
    • +6 more
    Oct 26, 2023

    Hemophilia A Trial in Tianjin (Single dose intravenous injection of BBM 002)

    Not yet recruiting
    • Hemophilia A
    • Single dose intravenous injection of BBM 002
    • Tianjin, Tianjin, China
      Institute of Hematology & Blood Diseases Hospital
    Jul 7, 2022

    Wiskott-Aldrich Syndrome (WAS) Trial in Milan (OTL-103)

    Active, not recruiting
    • Wiskott-Aldrich Syndrome (WAS)
    • OTL-103
    • Milan, Italy
      Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR
    Apr 21, 2022

    Infantile GM2 Gangliosidosis (Disorder) Trial in Kingston (TSHA-101)

    Active, not recruiting
    • Infantile GM2 Gangliosidosis (Disorder)
    • TSHA-101
    • Kingston, Ontario, Canada
      Queen's University/Kingston Health Sciences Centre
    Sep 12, 2022

    Friedreich Ataxia, Cardiomyopathy, Secondary Trial in United States (Low dose LX2006, High Dose LX2006)

    Recruiting
    • Friedreich Ataxia
    • Cardiomyopathy, Secondary
    • Low dose LX2006
    • High Dose LX2006
    • Los Angeles, California
    • +3 more
    Dec 2, 2022

    Study of Participants Previously Enrolled in the STARLIGHT Study

    Enrolling by invitation
    • Stargardt Disease
    • Gene Therapy Product-MCO-010
    • Miami, Florida
    • +1 more
    Sep 15, 2023

    Subjects Previously Enrolled in NCI Genitourinary Malignancies

    Enrolling by invitation
    • Cervical Intraepithelial Neoplasia
    • +4 more
      • Bethesda, Maryland
        National Institutes of Health Clinical Center
      Aug 20, 2022

      Homozygous Familial Hypercholesterolemia Trial in Xi'an (NGGT006)

      Not yet recruiting
      • Homozygous Familial Hypercholesterolemia
      • NGGT006
      • Xi'an, Shaanxi, China
        First Affiliated Hospital of Xian Jiaotong University
      Nov 5, 2023