Article ID | Journal | Published Year | Pages | File Type |
---|---|---|---|---|
8952436 | Ophthalmology | 2018 | 10 Pages |
Abstract
As a proof of concept, our results suggest that the ablate-and-replace strategy can ameliorate disease progression as measured by photoreceptor structure and function for both of the human mutation knock-in models. These results demonstrate the potency of the ablate-and-replace strategy to treat RP caused by different Rho mutations. Furthermore, because ablate-and-replace treatment is mutation independent, this strategy may be used to treat a wide array of dominant diseases in ophthalmology and other fields. Clinical trials using ablate-and-replace gene therapy would allow researchers to determine if this strategy provides any benefits for patients with diseases of interest.
Keywords
PBSCRISPRRetinitis pigmentosaADRPONLAAVgRNANHEJshort interfering RNAsiRNAclustered regularly interspaced short palindromic repeatbase pairguide RNAautosomal dominant retinitis pigmentosaRhoRhodopsinouter nuclear layerPhosphate-buffered salinepolymerase chain reactionPCRAdeno-associated virusNonhomologous end joining
Related Topics
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Medicine and Dentistry
Ophthalmology
Authors
Yi-Ting MS, MPhil, Wen-Hsuan MS, Ting-Ting MS, Wei-Pu MS, Christine L. BS, Karen S. BA, Xuan MD, Sally BS, Chyuan-Sheng PhD, Ruben BS, Pei-Yin MS, Stephen H. MD, PhD,