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Mechanistic and Therapeutic Insights into Ataxic Disorders with Pentanucleotide Expansions

Pentanucleotide expansion diseases constitute a special class of neurodegeneration. The repeat expansions occur in non-coding regions, have likely arisen from <i>Alu</i> elements, and often result in autosomal dominant or recessive phenotypes with underlying cerebellar neuropathology. When transcrib...

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Autori principali: Nan Zhang, Tetsuo Ashizawa
Natura: Artigo
Lingua:Inglês
Pubblicazione: MDPI AG 2022-05-01
Serie:Cells
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Accesso online:https://www.mdpi.com/2073-4409/11/9/1567
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