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

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

Inherited retinal dystrophies caused by dominant mutations in photoreceptor (PR) cell expressed genes are a major cause of irreversible vision loss. Oligonucleotide therapy has been of interest in diseases that conventional medicine cannot target. In the early days, small interfering RNAs (siRNAs) w...

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Detaylı Bibliyografya
Asıl Yazarlar: Shun-Yun Cheng, Jillian Caiazzi, Annabelle Biscans, Julia F. Alterman, Dimas Echeverria, Nicholas McHugh, Matthew Hassler, Samson Jolly, Delaney Giguere, Joris Cipi, Anastasia Khvorova, Claudio Punzo
Materyal Türü: Makale
Dil:English
Baskı/Yayın Bilgisi: Elsevier 2024-03-01
Seri Bilgileri:Molecular Therapy: Nucleic Acids
Konular:
MT: Oligonucleotides: Therapies and Applications
siRNA
photoreceptors
gene silencing
IRD
inherited retinal dystrophy
Online Erişim:http://www.sciencedirect.com/science/article/pii/S2162253123003062

Internet

http://www.sciencedirect.com/science/article/pii/S2162253123003062

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