Leber Congenital Amaurosis
Last Posted: Sep 21, 2021
- Gene therapy: perspectives from young adults with Leber's congenital amaurosis.
Napier Melanie P et al. Eye (London, England) 2021
- 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)
- Perspectives of people with inherited retinal diseases on ocular gene therapy in Australia: protocol for a national survey.
Mack Heather G et al. BMJ open 2021 11(6) e048361
- Safety and improved efficacy signals following gene therapy in childhood blindness caused by GUCY2D mutations.
Jacobson Samuel G et al. iScience 2021 24(5) 102409
- Molecular genetics with clinical characteristics of Leber congenital amaurosis in the Han population of western China.
Zhu Luyao et al. Ophthalmic genetics 2021 1-10
- Gene therapy for inherited retinal diseases: progress and possibilities.
Hu Monica L et al. Clinical & experimental optometry 2021 1-11
- 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
- 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
- Molecular and phenotypic investigation of a New Zealand cohort of childhood-onset retinal dystrophy.
Hull Sarah et al. American journal of medical genetics. Part C, Seminars in medical genetics 2020 Aug
- 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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