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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Detalles Bibliográficos
Autores principales: Alsing, Sidsel, Doktor, Thomas Koed, Askou, Anne Louise, Jensen, Emilie Grarup, Ahmadov, Ulvi, Kristensen, Lasse Sommer, Andresen, Brage Storstein, Aagaard, Lars, Corydon, Thomas J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8933642/
https://www.ncbi.nlm.nih.gov/pubmed/35356684
http://dx.doi.org/10.1016/j.omtn.2022.02.019

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8933642/
https://www.ncbi.nlm.nih.gov/pubmed/35356684
http://dx.doi.org/10.1016/j.omtn.2022.02.019

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