Antisense oligonucleotides

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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Main Authors: Daniel R. Scoles, Eric Vallabh Minikel, Stefan M. Pulst
格式: Revisão
语言:英语
出版: 2019
在线阅读:https://doi.org/10.1212/nxg.0000000000000323
https://ng.neurology.org/content/nng/5/2/e323.full.pdf
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