Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy

The development of antisense oligonucleotide therapy is an important advance in the identification of corrective therapy for neuromuscular diseases, such as spinal muscular atrophy (SMA). Because of difficulties of delivering single-stranded oligonucleotides to the CNS, current approaches have been...

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Detalles Bibliográficos
Publicado en:Proc Natl Acad Sci U S A
Main Authors: Hammond, Suzan M., Hazell, Gareth, Shabanpoor, Fazel, Saleh, Amer F., Bowerman, Melissa, Sleigh, James N., Meijboom, Katharina E., Zhou, Haiyan, Muntoni, Francesco, Talbot, Kevin, Gait, Michael J., Wood, Matthew J. A.
Formato: Artigo
Idioma:Inglês
Publicado: National Academy of Sciences 2016
Assuntos:
Biological Sciences
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC5047168/
https://ncbi.nlm.nih.gov/pubmed/27621445
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.1605731113
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