Druggable genome screen identifies new regulators of the abundance and toxicity of ATXN3, the Spinocerebellar Ataxia type 3 disease protein

Druggable genome screen identifies new regulators of the abundance and toxicity of ATXN3, the Spinocerebellar Ataxia type 3 disease protein

Spinocerebellar Ataxia type 3 (SCA3, also known as Machado-Joseph disease) is a neurodegenerative disorder caused by a CAG repeat expansion encoding an abnormally long polyglutamine (polyQ) tract in the disease protein, ataxin-3 (ATXN3). No preventive treatment is yet available for SCA3. Because SCA...

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Bibliographic Details
Main Authors: Naila S. Ashraf, Joanna R. Sutton, Yemen Yang, Bedri Ranxhi, Kozeta Libohova, Emily D. Shaw, Anna J. Barget, Sokol V. Todi, Henry L. Paulson, Maria do Carmo Costa
Format: Article
Language:English
Published: Elsevier 2020-04-01
Series:Neurobiology of Disease
Subjects:
Polyglutamine
Spinocerebellar ataxia
Machado-Joseph disease
Neurodegeneration
High-throughput screen
Human embryonic stem cells
Online Access:http://www.sciencedirect.com/science/article/pii/S0969996119303729

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http://www.sciencedirect.com/science/article/pii/S0969996119303729

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