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Gene-Delivery

Showing 1 - 25 of >10,000
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Duchenne Muscular Dystrophy Trial (delandistrogene moxeparvovec)

Not yet recruiting
  • Duchenne Muscular Dystrophy
  • delandistrogene moxeparvovec
  • (no location specified)
Nov 8, 2023

Limb-Girdle Muscular Dystrophy, Type 2E Trial in Columbus (SRP-9003)

Active, not recruiting
  • Limb-Girdle Muscular Dystrophy, Type 2E
  • SRP-9003
  • Columbus, Ohio
    Nationwide Children's Hospital
Sep 28, 2022

Duchenne Muscular Dystrophy Trial in Columbus (SRP-9001)

Active, not recruiting
  • Duchenne Muscular Dystrophy
  • SRP-9001
  • Columbus, Ohio
    Nationwide Children's Hospital
Jul 4, 2022

Sensorineural Hearing Loss, Bilateral Trial in Philadelphia (AAVAnc80-hOTOF via Akouos Delivery Device)

Recruiting
  • Sensorineural Hearing Loss, Bilateral
  • AAVAnc80-hOTOF via Akouos Delivery Device
  • Philadelphia, Pennsylvania
    Children's Hospital of Philadelphia
Apr 18, 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

SMARD1, CMT2S Trial in Columbus (Gene Therapy)

Enrolling by invitation
  • SMARD1
  • CMT2S
  • Gene Therapy
  • Columbus, Ohio
    Nationwide Children's Hospital
Dec 16, 2021

Muscular Dystrophy, Duchenne Trial in United States (SRP-9001)

Enrolling by invitation
  • Muscular Dystrophy, Duchenne
  • SRP-9001
  • Palo Alto, California
  • +4 more
Sep 28, 2022

Duchenne Muscular Dystrophy Trial in Columbus (scAAV9.U7.ACCA)

Active, not recruiting
  • Duchenne Muscular Dystrophy
  • scAAV9.U7.ACCA
  • Columbus, Ohio
    Nationwide Children's Hospital
Nov 25, 2021

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

Mesial Temporal Lobe Epilepsy Trial in Columbus (AAV9-hSyn1-miGRIK2)

Not yet recruiting
  • Mesial Temporal Lobe Epilepsy
  • AAV9-hSyn1-miGRIK2
  • Columbus, Ohio
    Ohio State University
Sep 25, 2023

Duchenne Muscular Dystrophy Trial in Worldwide (SRP-9001, Placebo)

Recruiting
  • Duchenne Muscular Dystrophy
  • SRP-9001
  • Placebo
  • Sacramento, California
  • +9 more
Mar 10, 2022

Canavan Disease Trial in Gainesville (rAAV9-CB6-AspA)

Available
  • Canavan Disease
  • Gainesville, Florida
    University of Florida
Apr 7, 2022

Alzheimer's Disease Trial in San Diego, Cleveland (AAV2-BDNF Gene Therapy)

Recruiting
  • Alzheimer's Disease
  • AAV2-BDNF Gene Therapy
  • San Diego, California
  • +1 more
Sep 1, 2021

Type 1 Diabetes Risk in Children During Routine Care Delivery

Recruiting
  • Type 1 Diabetes
  • Celiac Disease
  • Sera and whole blood sampling
  • Differential Gene Expression (DGE)
  • Bemidji, Minnesota
  • +3 more
Jan 18, 2022

Cancer Gene Mutation Trial in Bologna (Post-test genetic counseling delivered by telephone)

Recruiting
  • Cancer Gene Mutation
  • Post-test genetic counseling delivered by telephone
  • Bologna, Italy
    UO Genetica Medica, IRCCS AOU di Bologna
Jul 22, 2021

Neovascular Age-related Macular Degeneration, Wet Macular Degeneration, Wet Age-related Macular Degeneration Trial in United

Recruiting
  • Neovascular Age-related Macular Degeneration
  • +2 more
  • RGX-314
  • Phoenix, Arizona
  • +12 more
Oct 12, 2021

Alzheimer's Disease Trial in United States (CERE-110: Adeno-Associated Virus injection Surgery, Placebo Surgery)

Completed
  • Alzheimer's Disease
  • CERE-110: Adeno-Associated Virus injection Surgery
  • Placebo Surgery
  • Birmingham, Alabama
  • +9 more
Dec 14, 2020

Preterm Birth, Mother-Infant Interaction, Infant Development Trial in Cologne (skin-to-skin contact)

Recruiting
  • Preterm Birth
  • +2 more
  • skin-to-skin contact
  • Cologne, Northrhine-westfalia, Germany
    University hospital of Cologne, Department of Neonatology
Aug 4, 2023

COVID-19 on Placental Gene Expression and Pathology

Active, not recruiting
  • COVID-19
  • SARS-CoV-2 Infection
  • Positive for SARS-CoV-2 infection
  • Greenville, South Carolina
    Prisma Health Upstate
Jun 1, 2022

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

P47-Phox, Deficiency of Trial in London (Lentiviral vector transduced CD34+ cells)

Not yet recruiting
  • P47-Phox, Deficiency of
  • Lentiviral vector transduced CD34+ cells
  • London, United Kingdom
    Great Ormond Street Hospital
Aug 2, 2022

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

Duchenne Muscular Dystrophy Trial in United States (SRP-9001, Placebo)

Not yet recruiting
  • Duchenne Muscular Dystrophy
  • SRP-9001
  • Placebo
  • Little Rock, Arkansas
  • +13 more
May 19, 2023

Familial Hypercholesterolemia Trial in Xi'an (Low Density Lipoprotein Receptor mRNA Exosomes)

Not yet recruiting
  • Familial Hypercholesterolemia
  • Low Density Lipoprotein Receptor mRNA Exosomes
  • Xi'an, Shannxi, China
    Tangdu Hospital, Air Force Medical University
Sep 3, 2021

Parkinson's Disease Trial in San Francisco, Chicago (CERE-120: AAV2-NTN)

Completed
  • Parkinson's Disease
  • CERE-120: AAV2-NTN
  • San Francisco, California
  • +1 more
Nov 8, 2022