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Antisense oligonucleotides: A primer

There are few disease-modifying therapeutics for neurodegenerative diseases, but successes on the development of antisense oligonucleotide (ASO) therapeutics for spinal muscular atrophy and Duchenne muscular dystrophy predict a robust future for ASOs in medicine. Indeed, existing pipelines for the d...

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Detalles Bibliográficos
Publicado en:Neurol Genet
Main Authors: Scoles, Daniel R., Minikel, Eric V., Pulst, Stefan M.
Formato: Artigo
Idioma:Inglês
Publicado: Wolters Kluwer 2019
Assuntos:
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC6501637/
https://ncbi.nlm.nih.gov/pubmed/31119194
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1212/NXG.0000000000000323
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