Identification of a Peptide for Systemic Brain Delivery of a Morpholino Oligonucleotide in Mouse Models of Spinal Muscular Atrophy

Splice-switching antisense oligonucleotides are emerging treatments for neuromuscular diseases, with several splice-switching oligonucleotides (SSOs) currently undergoing clinical trials such as for Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA). However, the development of syst...

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Vydáno v:Nucleic Acid Ther
Hlavní autoři: Shabanpoor, Fazel, Hammond, Suzan M, Abendroth, Frank, Hazell, Gareth, Wood, Matthew J.A., Gait, Michael J.
Médium: Artigo
Jazyk:Inglês
Vydáno: Mary Ann Liebert, Inc. 2017
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Original Articles
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC5467147/
https://ncbi.nlm.nih.gov/pubmed/28118087
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/nat.2016.0652
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