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  2. Gene therapy clinical trials
This Panel is marked as Internal

Gene therapy clinical trials (Version 0.12)

Level 3: Clinical trials
Level 2: Actionable information

Panel types: Actionable
Description
This panel was made internal in September 2025 as it is now 7 years out of date and there are no plans to maintain it, making it a less useful/reliable resource. 

This panel was created from information curated from clinicaltrials.gov, searching for trials related the 'gene therapy' within the UK on 2nd May 2018. 

- Gene information was obtained from information within the trial description, or matching of the protein to the encoding gene. 
- Phenotype information is the disease provided within the trial information.
Panel Activity

3 reviewers

  • Ellen McDonagh (Genomics England Curator)

    Group: Other
    Workplace: Other

  • Eleanor Williams (Genomics England Curator)

    Group: Other
    Workplace: Other

  • vardha ismail (Wessex Regional Genetics Service)

    Group: NHS Genomic Medicine Centre
    Workplace: NHS clinical service

22 Entities

22 reviewed, 22 green

List Entity Reviews Mode of inheritance Details
22 Entitiess
Green List (high evidence)
ABCD1
1 review
1 green 		Not set
Sources
  • ClinicalTrials.gov
  • Expert Review Green
Phenotypes
  • Cerebral Adrenoleukodystrophy (CALD)
Tags
Green List (high evidence)
ADA
1 review
1 green 		Not set
Sources
  • ClinicalTrials.gov
  • Expert Review Green
Phenotypes
  • Adenosine deaminase (ADA) deficiency
Tags
Green List (high evidence)
ATP2A2
1 review
1 green 		Not set
Sources
  • ClinicalTrials.gov
  • Expert Review Green
Phenotypes
  • Chronic Heart Failure
  • Patients That Have Received a Left Ventricular Assist Device
Tags
Green List (high evidence)
CFTR
1 review
1 green 		Not set
Sources
  • ClinicalTrials.gov
  • Expert Review Green
Phenotypes
  • Cystic Fibrosis
Tags
Green List (high evidence)
CHM
1 review
1 green 		Not set
Sources
  • ClinicalTrials.gov
  • Expert Review Green
Phenotypes
  • Choroideraemia
Tags
Green List (high evidence)
CNGB3
1 review
1 green 		Not set
Sources
  • ClinicalTrials.gov
  • Expert Review Green
Phenotypes
  • Achromatopsia
Tags
Green List (high evidence)
CYBB
1 review
1 green 		Not set
Sources
  • ClinicalTrials.gov
  • Expert Review Green
Phenotypes
  • Granulomatous Disease, Chronic, X-linked
Tags
Green List (high evidence)
DDC
2 reviews
 BIALLELIC, autosomal or pseudoautosomal
Sources
  • Expert Review Green
  • Other
Tags
Green List (high evidence)
DMD
1 review
1 green 		Not set
Sources
  • ClinicalTrials.gov
  • Expert Review Green
Phenotypes
  • Duchenne Muscular Dystrophy
Tags
  • Skewed X-inactivation
Green List (high evidence)
F8
1 review
1 green 		Not set
Sources
  • ClinicalTrials.gov
  • Expert Review Green
Phenotypes
  • Haemophilia A
  • Severe Haemophilia A
Tags
Green List (high evidence)
F9
1 review
1 green 		Not set
Sources
  • ClinicalTrials.gov
  • Expert Review Green
Phenotypes
  • Hemophilia B
Tags
Green List (high evidence)
HBB
1 review
1 green 		Not set
Sources
  • ClinicalTrials.gov
  • Expert Review Green
Phenotypes
  • Beta-Thalassemia
Tags
Green List (high evidence)
IL2RG
1 review
1 green 		Not set
Sources
  • ClinicalTrials.gov
  • Expert Review Green
Phenotypes
  • X-linked Severe Combined Immunodeficiency
Tags
Green List (high evidence)
MT-ND4
1 review
1 green 		Not set
Sources
  • ClinicalTrials.gov
  • Expert Review Green
Phenotypes
  • Optic, Atrophy, Hereditary, Leber
Tags
Green List (high evidence)
OTC
1 review
1 green 		Not set
Sources
  • ClinicalTrials.gov
  • Expert Review Green
Phenotypes
  • Late-Onset OTC Deficiency
Tags
Green List (high evidence)
RPE65
1 review
1 green 		Not set
Sources
  • ClinicalTrials.gov
  • Expert Review Green
Phenotypes
  • Leber Congenital Amaurosis
  • Leber Hereditary Optic Neuropathy
  • Eye Diseases
  • Eye Diseases, Hereditary
  • Retinal Diseases
  • Retinal Degeneration
Tags
Green List (high evidence)
RPGR
1 review
1 green 		Not set
Sources
  • ClinicalTrials.gov
  • Expert Review Green
Phenotypes
  • X-Linked Retinitis Pigmentosa
Tags
Green List (high evidence)
SMN1
1 review
1 green 		Not set
Sources
  • ClinicalTrials.gov
  • Expert Review Green
Phenotypes
  • Spinal Muscular Atrophy Type 1
Tags
Green List (high evidence)
SMN2
1 review
1 green 		Not set
Sources
  • ClinicalTrials.gov
  • Expert Review Green
Phenotypes
  • {Spinal muscular atrophy, type III, modifier of}, OMIM:253400
Tags
Green List (high evidence)
SPINK5
1 review
1 green 		Not set
Sources
  • ClinicalTrials.gov
  • Expert Review Green
Phenotypes
  • Netherton syndrome
Tags
Green List (high evidence)
UGT1A1
1 review
1 green 		Not set
Sources
  • ClinicalTrials.gov
  • Expert Review Green
Phenotypes
  • Crigler-Najjar Syndrome
Tags
Green List (high evidence)
WAS
1 review
1 green 		Not set
Sources
  • ClinicalTrials.gov
  • Expert Review Green
Phenotypes
  • Wiskott Aldrich syndrome
Tags

Downloads

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  • Green list (high evidence)
  • Green and Amber Genes
  • Amber Genes
  • Red list (low evidence)

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