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dc.contributor.authorPetit, Lolita
dc.contributor.authorPunzo, Claudio
dc.date2022-08-11T08:09:46.000
dc.date.accessioned2022-08-23T16:42:39Z
dc.date.available2022-08-23T16:42:39Z
dc.date.issued2016-10-01
dc.date.submitted2017-01-09
dc.identifier.citationDiscov Med. 2016 Oct;22(121):221-229.
dc.identifier.issn1539-6509 (Linking)
dc.identifier.pmid27875674
dc.identifier.urihttp://hdl.handle.net/20.500.14038/40137
dc.description.abstractThere is an impelling need to develop effective therapeutic strategies for patients with retinal disorders. Gleaning from the large quantity of information gathered over the past two decades on the mechanisms governing degeneration of the retina, it is now possible to devise innovative therapies based on retinal gene transfer. Different gene-based approaches are under active investigation. They include strategies to correct the specific genetic defect in inherited retinal diseases, strategies to delay the onset of blindness independently of the disease-causing mutations, and strategies to reactivate residual cells at late stages of the diseases. In this review, we discuss the status of application of these technologies, outlining the future therapeutic potential for many forms of retinal blinding diseases.
dc.language.isoen_US
dc.relation<a href="http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?db=pubmed&cmd=Retrieve&list_uids=27875674&dopt=Abstract">Link to Article in PubMed</a>
dc.relation.urlhttps://www.ncbi.nlm.nih.gov/pmc/articles/PMC5142441/
dc.subjectEye Diseases
dc.subjectGenetics and Genomics
dc.subjectOphthalmology
dc.titleGene therapy approaches for the treatment of retinal disorders
dc.typeJournal Article
dc.source.journaltitleDiscovery medicine
dc.source.volume22
dc.source.issue121
dc.identifier.legacycoverpagehttps://escholarship.umassmed.edu/oapubs/2937
dc.identifier.contextkey9532576
html.description.abstract<p>There is an impelling need to develop effective therapeutic strategies for patients with retinal disorders. Gleaning from the large quantity of information gathered over the past two decades on the mechanisms governing degeneration of the retina, it is now possible to devise innovative therapies based on retinal gene transfer. Different gene-based approaches are under active investigation. They include strategies to correct the specific genetic defect in inherited retinal diseases, strategies to delay the onset of blindness independently of the disease-causing mutations, and strategies to reactivate residual cells at late stages of the diseases. In this review, we discuss the status of application of these technologies, outlining the future therapeutic potential for many forms of retinal blinding diseases.</p>
dc.identifier.submissionpathoapubs/2937
dc.contributor.departmentGene Therapy Center
dc.contributor.departmentDepartment of Ophthalmology
dc.source.pages221-229


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