Development of an AAV-Based MicroRNA Gene Therapy to Treat Machado-Joseph Disease

Spinocerebellar ataxia type 3 (SCA3), or Machado-Joseph disease (MJD), is a progressive neurodegenerative disorder caused by a CAG expansion in the ATXN3 gene. The expanded CAG repeat is translated into a prolonged polyglutamine repeat in the ataxin-3 protein and accumulates within inclusions, acqui...

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Vydáno v:Mol Ther Methods Clin Dev
Hlavní autoři: Martier, Raygene, Sogorb-Gonzalez, Marina, Stricker-Shaver, Janice, Hübener-Schmid, Jeannette, Keskin, Sonay, Klima, Jiri, Toonen, Lodewijk J., Juhas, Stefan, Juhasova, Jana, Ellederova, Zdenka, Motlik, Jan, Haas, Eva, van Deventer, Sander, Konstantinova, Pavlina, Nguyen, Huu Phuc, Evers, Melvin M.
Médium: Artigo
Jazyk:Inglês
Vydáno: American Society of Gene & Cell Therapy 2019
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On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC6889651/
https://ncbi.nlm.nih.gov/pubmed/31828177
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2019.10.008
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