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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Dades bibliogràfiques
Publicat a:Proc Natl Acad Sci U S A
Autors principals: 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.
Format: Artigo
Idioma:Inglês
Publicat: National Academy of Sciences 2016
Matèries:
Biological Sciences
Accés en línia: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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