Assisted delivery of antisense therapeutics in animal models of heritable neurodegenerative and neuromuscular disorders: a systematic review and meta-analysis

Antisense oligonucleotide (AON)-based therapies hold promise for a range of neurodegenerative and neuromuscular diseases and have shown benefit in animal models and patients. Success in the clinic is nevertheless still limited, due to unfavourable biodistribution and poor cellular uptake of AONs. Ex...

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Detalles Bibliográficos
Autores principales: van der Bent, M. Leontien, Paulino da Silva Filho, Omar, van Luijk, Judith, Brock, Roland, Wansink, Derick G.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2018
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5843643/
https://www.ncbi.nlm.nih.gov/pubmed/29520012
http://dx.doi.org/10.1038/s41598-018-22316-7

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5843643/
https://www.ncbi.nlm.nih.gov/pubmed/29520012
http://dx.doi.org/10.1038/s41598-018-22316-7

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