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Cas-Based Systems for RNA Editing in Gene Therapy of Monogenic Diseases: In Vitro and in Vivo Application and Translational Potential

Rare genetic diseases reduce quality of life and can significantly shorten the lifespan. There are few effective treatment options for these diseases, and existing therapeutic strategies often represent only supportive or palliative care. Therefore, designing genetic-engineering technologies for the...

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Detalles Bibliográficos
Autores principales: Reshetnikov, Vasiliy V., Chirinskaite, Angelina V., Sopova, Julia V., Ivanov, Roman A., Leonova, Elena I.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9245891/
https://www.ncbi.nlm.nih.gov/pubmed/35784464
http://dx.doi.org/10.3389/fcell.2022.903812