PanelApp
  1. Panels
  2. Gene therapy clinical trials
Description
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

1 reviewer

  • Ellen McDonagh (Genomics England Curator)

    Group: Other
    Workplace: Other

21 Entities

21 reviewed, 21 green

List Entity Reviews Mode of inheritance Details
21 Entitiess
Green Green List (high evidence)
ABCD1
1 review
1 green 		Not set
Sources
  • ClinicalTrials.gov
  • Expert Review Green
Phenotypes
  • Cerebral Adrenoleukodystrophy (CALD)
Tags
Green Green List (high evidence)
ADA
1 review
1 green 		Not set
Sources
  • ClinicalTrials.gov
  • Expert Review Green
Phenotypes
  • Adenosine deaminase (ADA) deficiency
Tags
Green 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 Green List (high evidence)
CFTR
1 review
1 green 		Not set
Sources
  • ClinicalTrials.gov
  • Expert Review Green
Phenotypes
  • Cystic Fibrosis
Tags
Green Green List (high evidence)
CHM
1 review
1 green 		Not set
Sources
  • ClinicalTrials.gov
  • Expert Review Green
Phenotypes
  • Choroideraemia
Tags
Green Green List (high evidence)
CNGB3
1 review
1 green 		Not set
Sources
  • ClinicalTrials.gov
  • Expert Review Green
Phenotypes
  • Achromatopsia
Tags
Green 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 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 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 Green List (high evidence)
F9
1 review
1 green 		Not set
Sources
  • ClinicalTrials.gov
  • Expert Review Green
Phenotypes
  • Hemophilia B
Tags
Green Green List (high evidence)
HBB
1 review
1 green 		Not set
Sources
  • ClinicalTrials.gov
  • Expert Review Green
Phenotypes
  • Beta-Thalassemia
Tags
Green 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 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 Green List (high evidence)
OTC
1 review
1 green 		Not set
Sources
  • ClinicalTrials.gov
  • Expert Review Green
Phenotypes
  • Late-Onset OTC Deficiency
Tags
Green 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 Green List (high evidence)
RPGR
1 review
1 green 		Not set
Sources
  • ClinicalTrials.gov
  • Expert Review Green
Phenotypes
  • X-Linked Retinitis Pigmentosa
Tags
Green 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 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 Green List (high evidence)
SPINK5
1 review
1 green 		Not set
Sources
  • ClinicalTrials.gov
  • Expert Review Green
Phenotypes
  • Netherton syndrome
Tags
Green Green List (high evidence)
UGT1A1
1 review
1 green 		Not set
Sources
  • ClinicalTrials.gov
  • Expert Review Green
Phenotypes
  • Crigler-Najjar Syndrome
Tags
Green Green List (high evidence)
WAS
1 review
1 green 		Not set
Sources
  • ClinicalTrials.gov
  • Expert Review Green
Phenotypes
  • Wiskott Aldrich syndrome
Tags

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