VEGFA-targeting miR-agshRNAs combine efficacy with specificity and safety for retinal gene therapy

VEGFA-targeting miR-agshRNAs combine efficacy with specificity and safety for retinal gene therapy

Retinal gene therapy using RNA interference (RNAi) to silence targeted genes requires both efficacy and safety. Short hairpin RNAs (shRNAs) are useful for RNAi, but high expression levels and activity from the co-delivered passenger strand may cause undesirable cellular responses. Ago2-dependent shR...

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Bibliographic Details
Main Authors: Sidsel Alsing, Thomas Koed Doktor, Anne Louise Askou, Emilie Grarup Jensen, Ulvi Ahmadov, Lasse Sommer Kristensen, Brage Storstein Andresen, Lars Aagaard, Thomas J. Corydon
Format: Article
Language:English
Published: Elsevier 2022-06-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
Non-coding RNAs
Dicer-independent shRNAs
Pol II-driven miRNA scaffold
RNA interference
agshRNA
agoshRNA
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253122000476

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http://www.sciencedirect.com/science/article/pii/S2162253122000476

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