Therapeutic Strategies for Spinocerebellar Ataxia Type 1.

Kerkhof, Laurie M C; van de Warrenburg, Bart P C; van Roon-Mom, Willeke M C; Buijsen, Ronald A M

Kerkhof, Laurie M C; van de Warrenburg, Bart P C; van Roon-Mom, Willeke M C; Buijsen, Ronald A M.

Afiliación

Kerkhof LMC; Department of Human Genetics, Leiden University Medical Center, 2333 ZA Leiden, The Netherlands.
van de Warrenburg BPC; Dutch Center for RNA Therapeutics, Leiden University Medical Center, 2333 ZA Leiden, The Netherlands.
van Roon-Mom WMC; Department of Neurology, Donders Institute for Brain, Cognition, and Behaviour, Radboud University Medical Center, 6525 GA Nijmegen, The Netherlands.
Buijsen RAM; Department of Human Genetics, Leiden University Medical Center, 2333 ZA Leiden, The Netherlands.

Biomolecules ; 13(5)2023 05 02.

Article en En | MEDLINE | ID: mdl-37238658

Asunto(s)

Cerebelo; Ataxias Espinocerebelosas; Humanos; Cerebelo/metabolismo; Ataxias Espinocerebelosas/genética; Ataxias Espinocerebelosas/terapia; Ataxina-1/genética; Ataxina-1/metabolismo; Células de Purkinje/patología

Palabras clave

ATXN1; Spinocerebellar ataxia type 1; ataxin1; clinical trials; gain-of-function mechanism; neurodegenerative disorder; polyQ disorders; preclinical research; therapeutic strategies

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Texto completo: 1 Base de datos: MEDLINE Asunto principal: Cerebelo / Ataxias Espinocerebelosas Idioma: En Revista: Biomolecules Año: 2023 Tipo del documento: Article

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PubMed Links

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Texto completo: 1 Base de datos: MEDLINE Asunto principal: Cerebelo / Ataxias Espinocerebelosas Idioma: En Revista: Biomolecules Año: 2023 Tipo del documento: Article