Translational potential of base-editing tools for gene therapy of monogenic diseases

Millions of people worldwide have rare genetic diseases that are caused by various mutations in DNA sequence. Classic treatments of rare genetic diseases are often ineffective, and therefore great hopes are placed on gene-editing methods. A DNA base–editing system based on nCas9 (Cas9 with a nickase...

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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:
Bioengineering and Biotechnology
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9399415/
https://www.ncbi.nlm.nih.gov/pubmed/36032737
http://dx.doi.org/10.3389/fbioe.2022.942440

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9399415/
https://www.ncbi.nlm.nih.gov/pubmed/36032737
http://dx.doi.org/10.3389/fbioe.2022.942440

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