Delivery is key: lessons learnt from developing splice‐switching antisense therapies

The use of splice‐switching antisense therapy is highly promising, with a wealth of pre‐clinical data and numerous clinical trials ongoing. Nevertheless, its potential to treat a variety of disorders has yet to be realized. The main obstacle impeding the clinical translation of this approach is the...

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Detalles Bibliográficos
Autores principales: Godfrey, Caroline, Desviat, Lourdes R, Smedsrød, Bård, Piétri‐Rouxel, France, Denti, Michela A, Disterer, Petra, Lorain, Stéphanie, Nogales‐Gadea, Gisela, Sardone, Valentina, Anwar, Rayan, EL Andaloussi, Samir, Lehto, Taavi, Khoo, Bernard, Brolin, Camilla, van Roon‐Mom, Willeke MC, Goyenvalle, Aurélie, Aartsma‐Rus, Annemieke, Arechavala‐Gomeza, Virginia
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2017
Materias:
Reviews
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5412803/
https://www.ncbi.nlm.nih.gov/pubmed/28289078
http://dx.doi.org/10.15252/emmm.201607199

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5412803/
https://www.ncbi.nlm.nih.gov/pubmed/28289078
http://dx.doi.org/10.15252/emmm.201607199

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