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Challenges to oligonucleotides-based therapeutics for Duchenne muscular dystrophy

Antisense oligonucleotides are short nucleic acids designed to bind to specific messenger RNAs in order to modulate splicing patterns or inhibit protein translation. As such, they represent promising therapeutic tools for many disorders and have been actively developed for more than 20 years as a fo...

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Autors principals: Goyenvalle, Aurélie, Davies, Kay E
Format: Artigo
Idioma:Inglês
Publicat: BioMed Central 2011
Matèries:
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC3156649/
https://ncbi.nlm.nih.gov/pubmed/21798085
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/2044-5040-1-8
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