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mRNA trans‐splicing in gene therapy for genetic diseases

Spliceosome‐mediated RNA trans‐splicing, or SMaRT, is a promising strategy to design innovative gene therapy solutions for currently intractable genetic diseases. SMaRT relies on the correction of mutations at the post‐transcriptional level by modifying the mRNA sequence. To achieve this, an exogeno...

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Detalles Bibliográficos
Autores principales:	Berger, Adeline, Maire, Séverine, Gaillard, Marie‐Claude, Sahel, José‐Alain, Hantraye, Philippe, Bemelmans, Alexis‐Pierre
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley & Sons, Inc. 2016
Materias:	Advanced Reviews
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5071737/ https://www.ncbi.nlm.nih.gov/pubmed/27018401 http://dx.doi.org/10.1002/wrna.1347

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