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

Author:

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.

Funder

European Community’s Seventh Framework Programme

National Sustainability Program I

Czech Ministry of Education, Youth and Sports

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference74 articles.

1. Machado-Joseph disease/spinocerebellar ataxia type 3: lessons from disease pathogenesis and clues into therapy;Matos;J. Neurochem.,2019

2. Autosomal dominant system degeneration in Portuguese families of the Azores Islands. A new genetic disorder involving cerebellar, pyramidal, extrapyramidal and spinal cord motor functions;Coutinho;Neurology,1978

3. Machado-Joseph disease: an autosomal dominant motor system degeneration;Rosenberg;Mov. Disord.,1992

4. Spinocerebellar ataxia type 1 and Machado-Joseph disease: incidence of CAG expansions among adult-onset ataxia patients from 311 families with dominant, recessive, or sporadic ataxia;Ranum;Am. J. Hum. Genet.,1995

5. Autosomal dominant cerebellar ataxias: clinical features, genetics, and pathogenesis;Schöls;Lancet Neurol.,2004

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