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Precision Health Database


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Rare Diseases

Last data update: Sep 29, 2023. (Total: 61659 Documents since 2012)
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Records 1 - 11 (of 11 Records)

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Update on Gene Therapy Clinical Trials for Choroideremia and Potential Experimental Therapies.
Abbouda Alessandro et al. Medicina (Kaunas, Lithuania) 2021 Jan 57(1)
Current Clinical Applications of in vivo Gene Therapy with AAVs.
Mendell Jerry R et al. Molecular therapy : the journal of the American Society of Gene Therapy 2020 Dec
Genetic testing for inherited eye conditions in over 6,000 individuals through the eyeGENE network.
Goetz Kerry E et al. American journal of medical genetics. Part C, Seminars in medical genetics 2020 Sep
Perspectives on Gene Therapy: Choroideremia Represents a Challenging Model for the Treatment of Other Inherited Retinal Degenerations.
MacDonald Ian M et al. Translational vision science & technology 2020 Feb 9(3) 17
Genetic architecture of inherited retinal degeneration in Germany: A large cohort study from a single diagnostic center over a 9-year period.
Weisschuh Nicole et al. Human mutation 2020 Jun
Choroideremia Gene Therapy Phase 2 Clinical Trial: 24-Month Results.
Lam Byron L et al. American journal of ophthalmology 2019 19765-73
Gene Therapy for Choroideremia-Progress and Remaining Questions.
Duncan Jacque L et al. JAMA ophthalmology 2019 Aug
Two-Year Results After AAV2-Mediated Gene Therapy for Choroideremia: The Alberta Experience.
Dimopoulos Ioannis S et al. American journal of ophthalmology 2018 193130-142
The Role of Gene Therapy in the Treatment of Retinal Diseases: A Review.
Campa C et al. Current gene therapy 2017 17(3) 194-213
Gene therapy for inherited retinal and optic nerve degenerations.
Moore Nicholas A et al. Expert opinion on biological therapy 2017 Oct 1-13
Clinical utility gene card for: choroideremia.
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