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Public Health Genomics and Precision Health Knowledge Base (v9.0)
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Precision Health Database|Search|Public Health Genomics and Precision Health Knowledge Base (PHGKB)
Rare Diseases
Last data update: Apr 25, 2024
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Leber Congenital Amaurosis[original query]>>Reviews/Commentaries[Product Type]
Gene therapy: perspectives from young adults with Leber's congenital amaurosis.
Napier Melanie P et al. Eye (London, England) 2021
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Inherited Retinal Diseases Due to RPE65 Variants: From Genetic Diagnostic Management to Therapy.
Aoun Manar et al. International journal of molecular sciences 2021 22(13)
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Gene therapy for inherited retinal diseases: progress and possibilities.
Hu Monica L et al. Clinical & experimental optometry 2021 1-11
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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
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Focused Update on AAV-Based Gene Therapy Clinical Trials for Inherited Retinal Degeneration.
Fuller-Carter Paula I et al. BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy 2020 Nov
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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
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Leber congenital amaurosis: Current genetic basis, scope for genetic testing and personalized medicine.
Kondkar Altaf A et al. Experimental eye research 2019 189107834
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CRISPR enters its first human clinical trials- The gene editor targets cancer, blood disorders and blindness
T Hesman, Science News, August 14, 2019
[Genetic diagnostics of retinal dystrophies : Breakthrough with new methods of DNA sequencing].
Bolz H J et al. Der Ophthalmologe : Zeitschrift der Deutschen Ophthalmologischen Gesellschaft 2018 Jul
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13-Year-Old Boy Is First Person in US to Receive Newly Approved Gene Therapy for Blindness
KV Brown, Gizmodo, Mar 22, 2018
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Page last reviewed:
Feb 1, 2024
Page last updated:
Apr 25, 2024
Content source:
Public Health Genomics Branch in the Division of Blood Disorders and Public Health Genomics
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National Center on Birth Defects and Developmental Disabilities
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