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A Single Transcript Knockdown-Replacement Strategy Employing 5’ UTR Secondary Structures to Precisely Titrate Rescue Protein Translation

One overarching goal of gene therapy is the replacement of faulty genes with functional ones. A significant hurdle is presented by the fact that under- or over-expression of a protein may cause disease as readily as coding mutations. There is a clear and present need for pipelines to translate exper...

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Detalles Bibliográficos
Autores principales: Millette, Matthew M., Holland, Elizabeth D., Tenpas, Tanner J., Dent, Erik W.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8995503/
https://www.ncbi.nlm.nih.gov/pubmed/35419562
http://dx.doi.org/10.3389/fgeed.2022.803375