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

• Main Authors: Sidsel Alsing (Author), Thomas Koed Doktor (Author), Anne Louise Askou (Author), Emilie Grarup Jensen (Author), Ulvi Ahmadov (Author), Lasse Sommer Kristensen (Author), Brage Storstein Andresen (Author), Lars Aagaard (Author), Thomas J. Corydon (Author)
• Format: Book
• Published: Elsevier, 2022-06-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...