Single intravitreal administration of a tetravalent siRNA exhibits robust and efficient gene silencing in mouse and pig photoreceptors.

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第一作者： Shun-Yun,Cheng

第一单位： Department of Ophthalmology and Visual Sciences, University of Massachusetts Chan Medical School, Worcester, MA 01605, USA.

作者： Shun-Yun,Cheng ^[1] ; Jillian,Caiazzi ^[2] ; Annabelle,Biscans ^[3] ; Julia F,Alterman ^[4] ; Dimas,Echeverria ^[4] ; Nicholas,McHugh ^[4] ; Matthew,Hassler ^[4] ; Samson,Jolly ^[4] ; Delaney,Giguere ^[4] ; Joris,Cipi ^[1] ; Anastasia,Khvorova ^[1] ; Claudio,Punzo ^[1]

作者单位： Department of Ophthalmology and Visual Sciences, University of Massachusetts Chan Medical School, Worcester, MA 01605, USA. ^[1] Horae Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, MA 01605, USA. ^[2] Department of Neurobiology, University of Massachusetts Chan Medical School, Worcester, MA 01605, USA. ^[3] RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, MA 01605, USA. ^[4]

关键词 IRD MT: Oligonucleotides: Therapies and Applications gene silencing inherited retinal dystrophy multivalency photoreceptors siRNA

DOI 10.1016/j.omtn.2023.102088

PMID 38192611

发布时间 2024-02-10

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