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Developing gene knockdown-replacement therapies for spinocerebellar ataxia type 7

Abstract:

For many dominant diseases, conventional treatment options are limited. This makes them attractive candidates for gene therapy, which may be directed to specifically silence a disease-causing allele. However, many mutations are not easily amenable to this technique, including nucleotide repeat expansions, which cause numerous neurodegenerative diseases such as Huntington’s disease and several Spinocerebellar Ataxias. Combined gene knockdown and replacement (K&R;) may present a more pra...

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Institution:
University of Oxford
Division:
MSD
Department:
Physiology Anatomy & Genetics
Oxford college:
Wolfson College
Role:
Author

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Role:
Supervisor
Publication date:
2013
Type of award:
DPhil
Level of award:
Doctoral
Awarding institution:
University of Oxford
Language:
English
Keywords:
Subjects:
UUID:
uuid:9e0570b6-f811-473a-a007-185fc8c77fc6
Local pid:
ora:10243
Deposit date:
2015-02-27

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