Last Posted: Jan 19, 2021
- 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
- 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
- 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
- Gene therapy for inherited retinal and optic nerve degenerations.
Moore Nicholas A et al. Expert opinion on biological therapy 2017 Oct 1-13
About Rare Diseases PHGKB
Rare Disease PHGKB is an online, continuously updated, searchable database of published scientific literature, CDC and NIH resources, and other information that address the public health impact and translation of genomic and other precision health discoveries into improved health outcomes related to rare diseases...more
Selected Rare Diseases
- Alpha-1 Antitrypsin Deficiency
- Amyotrophic Lateral Sclerosis
- Brugada Syndrome
- Cerebral Palsy
- Cystic Fibrosis
- Duchenne Muscular Dystrophy
- Erythema Multiforme
- Familial Mediterranean Fever
- Fragile X Syndrome
- Gaucher Disease
- Graves Disease
- Huntington Disease
- Myasthenia Gravis
- Retinitis Pigmentosa
- Severe Combined Immunodeficiency
Disclaimer: Articles listed in the Public Health Knowledge Base are selected by the CDC Office of Public Health Genomics to provide current awareness of the literature and news. Inclusion in the update does not necessarily represent the views of the Centers for Disease Control and Prevention nor does it imply endorsement of the article's methods or findings. CDC and DHHS assume no responsibility for the factual accuracy of the items presented. The selection, omission, or content of items does not imply any endorsement or other position taken by CDC or DHHS. Opinion, findings and conclusions expressed by the original authors of items included in the update, or persons quoted therein, are strictly their own and are in no way meant to represent the opinion or views of CDC or DHHS. References to publications, news sources, and non-CDC Websites are provided solely for informational purposes and do not imply endorsement by CDC or DHHS.
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- Page last updated:Jun 02, 2023
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