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The atlas of RNase H antisense oligonucleotide distribution and activity in the CNS of rodents and non-human primates following central administration

Antisense oligonucleotides (ASOs) have emerged as a new class of drugs to treat a wide range of diseases, including neurological indications. Spinraza, an ASO that modulates splicing of SMN2 RNA, has shown profound disease modifying effects in Spinal Muscular Atrophy (SMA) patients, energizing effor...

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Detalles Bibliográficos
Autores principales:	Jafar-nejad, Paymaan, Powers, Berit, Soriano, Armand, Zhao, Hien, Norris, Daniel A, Matson, John, DeBrosse-Serra, Beatrice, Watson, Jamie, Narayanan, Padmakumar, Chun, Seung J, Mazur, Curt, Kordasiewicz, Holly, Swayze, Eric E, Rigo, Frank
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Oxford University Press 2020
Materias:	Chemical Biology and Nucleic Acid Chemistry
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7826274/ https://www.ncbi.nlm.nih.gov/pubmed/33367834 http://dx.doi.org/10.1093/nar/gkaa1235

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