Antisense Oligonucleotides Reduce RNA Foci in Spinocerebellar Ataxia 36 Patient iPSCs

Spinocerebellar ataxia type 36 is a late-onset, slowly progressive cerebellar syndrome with motor neuron degeneration that is caused by expansions of a hexanucleotide repeat (GGCCTG) in the noncoding region of NOP56 gene, with a histopathological feature of RNA foci formation in postmortem tissues....

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Detalhes bibliográficos
Publicado no:Mol Ther Nucleic Acids
Main Authors: Matsuzono, Kosuke, Imamura, Keiko, Murakami, Nagahisa, Tsukita, Kayoko, Yamamoto, Takuya, Izumi, Yuishin, Kaji, Ryuji, Ohta, Yasuyuki, Yamashita, Toru, Abe, Koji, Inoue, Haruhisa
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2017
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5504081/
https://ncbi.nlm.nih.gov/pubmed/28918022
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2017.06.017
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