Mirtron-mediated RNA knockdown/replacement therapy for the treatment of dominant retinitis pigmentosa

Rhodopsin-related dominant retinitis pigmentosa is a degenerative disease of the retina of the eye for which there is no current treatment. In this study, the authors use a novel form of RNA-interference- artificial mirtrons- to slow retinal degeneration in a mouse model of the disease.

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Auteurs principaux: Harry O. Orlans, Michelle E. McClements, Alun R. Barnard, Cristina Martinez-Fernandez de la Camara, Robert E. MacLaren
Format: article
Langue:EN
Publié: Nature Portfolio 2021
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Accès en ligne:https://doaj.org/article/f079a2227d8741e1858d4c8fe8a57b28
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Résumé:Rhodopsin-related dominant retinitis pigmentosa is a degenerative disease of the retina of the eye for which there is no current treatment. In this study, the authors use a novel form of RNA-interference- artificial mirtrons- to slow retinal degeneration in a mouse model of the disease.