Selective suppression of polyglutamine-expanded protein by lipid nanoparticle-delivered siRNA targeting CAG expansions in the mouse CNS Article Swipe

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Tomoki Hirunagi , Kentaro Sahashi , Kiyoshi Tachikawa , Angel Leu , Michelle Nguyen , Rajesh Mukthavaram , Priya Karmali , Padmanabh Chivukula , Genki Tohnai , Madoka Iida , Kazunari Onodera , Manabu Ohyama , Yohei Okada , Hideyuki Okano , Masahisa Katsuno ·

YOU? · · 2021 · Open Access · · DOI: https://doi.org/10.1016/j.omtn.2021.02.007

Related Topics

Small Interfering Rna

Spinocerebellar Ataxia

Concepts

Spinal and bulbar muscular atrophy Trinucleotide repeat expansion Gene silencing Small interfering RNA Spinocerebellar ataxia Biology Mutant RNA interference Cancer research Molecular biology Genetics Gene Androgen receptor Transfection RNA Prostate cancer Cancer Allele

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Type: article
Language: en
Landing Page: https://doi.org/10.1016/j.omtn.2021.02.007
PDF: http://www.cell.com/article/S2162253121000421/pdf
OA Status: gold
Cited By: 27
References: 46
Related Works: 10
OpenAlex ID: https://openalex.org/W3129881290

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OpenAlex ID: https://openalex.org/W3129881290

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DOI: https://doi.org/10.1016/j.omtn.2021.02.007

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Title: Selective suppression of polyglutamine-expanded protein by lipid nanoparticle-delivered siRNA targeting CAG expansions in the mouse CNS

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Type: article

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Language: en

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Publication year: 2021

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Publication date: 2021-02-16

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Authors: Tomoki Hirunagi, Kentaro Sahashi, Kiyoshi Tachikawa, Angel Leu, Michelle Nguyen, Rajesh Mukthavaram, Priya Karmali, Padmanabh Chivukula, Genki Tohnai, Madoka Iida, Kazunari Onodera, Manabu Ohyama, Yohei Okada, Hideyuki Okano, Masahisa Katsuno

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Landing page: https://doi.org/10.1016/j.omtn.2021.02.007

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PDF URL: https://www.cell.com/article/S2162253121000421/pdf

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OA status: gold

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OA URL: https://www.cell.com/article/S2162253121000421/pdf

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Concepts: Spinal and bulbar muscular atrophy, Trinucleotide repeat expansion, Gene silencing, Small interfering RNA, Spinocerebellar ataxia, Biology, Mutant, RNA interference, Cancer research, Molecular biology, Genetics, Gene, Androgen receptor, Transfection, RNA, Prostate cancer, Cancer, Allele

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Cited by: 27

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Citations by year (recent): 2025: 6, 2024: 6, 2023: 7, 2022: 6, 2021: 2

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References (count): 46

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Related works (count): 10

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