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

Autor: Sidsel Alsing, Thomas Koed Doktor, Anne Louise Askou, Emilie Grarup Jensen, Ulvi Ahmadov, Lasse Sommer Kristensen, Brage Storstein Andresen, Lars Aagaard, Thomas J. Corydon

Publikováno v: Molecular Therapy: Nucleic Acids, Vol 28, Iss , Pp 58-76 (2022)

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

Externí odkaz: https://doaj.org/article/6de388c12e504aaf91ee9aa13a988490

Efficient Knockdown and Lack of Passenger Strand Activity by Dicer-Independent shRNAs Expressed from Pol II-Driven MicroRNA Scaffolds

Autor: Claudia R. Cecchi, Lars Aagaard, Erik Kaadt, Sidsel Alsing, Thomas J. Corydon, Christian Kroun Damgaard

Publikováno v: Kaadt, E, Alsing, S, Cecchi, C R, Damgaard, C K, Corydon, T J & Aagaard, L 2019, ' Efficient Knockdown and Lack of Passenger Strand Activity by Dicer-Independent shRNAs Expressed from Pol II-Driven MicroRNA Scaffolds ', Molecular Therapy-Nucleic Acids, vol. 14, pp. 318-328 . https://doi.org/10.1016/j.omtn.2018.11.013
Molecular Therapy. Nucleic Acids
Molecular Therapy: Nucleic Acids, Vol 14, Iss, Pp 318-328 (2019)

The expression of short hairpin RNAs (shRNAs) may result in unwanted activity from the co-processed passenger strand. Recent studies have shown that shortening the stem of conventional shRNAs abolishes passenger strand release. These Dicer-independen

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::bd1fc370a457137476f1763ea265d0d7
https://pure.au.dk/portal/da/publications/efficient-knockdown-and-lack-of-passenger-strand-activity-by-dicerindependent-shrnas-expressed-from-pol-iidriven-microrna-scaffolds(e4ae4aa8-4d5a-46b2-a3cf-5de53b7b176c).html