RNA Interference Therapy for Machado–Joseph Disease: Long-Term Safety Profile of Lentiviral Vectors Encoding Short Hairpin RNAs Targeting Mutant Ataxin-3-Reference-Cited by-同舟云学术

RNA Interference Therapy for Machado–Joseph Disease: Long-Term Safety Profile of Lentiviral Vectors Encoding Short Hairpin RNAs Targeting Mutant Ataxin-3

Published:2019-07 Issue:7 Volume:30 Page:841-854
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Nóbrega Clévio¹²³,Codêsso José Miguel¹,Mendonça Liliana¹,Pereira de Almeida Luís¹⁴

Affiliation:

1. Center for Neuroscience and Cell Biology, University of Coimbra, Coimbra, Portugal

2. Department of Biomedical Sciences and Medicine, University of Algarve, Faro, Portugal

3. Centre for Biomedical Research, University of Algarve, Faro, Portugal.

4. Faculty of Pharmacy, University of Coimbra, Coimbra, Portugal

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

https://www.liebertpub.com/doi/pdf/10.1089/hum.2018.157

Reference51 articles.

1. Trinucleotide expansion within the MJD1 gene presents clinically as spinocerebellar ataxia and occurs most frequently in German SCA patients

2. Spinocerebellar ataxia 3 and machado-joseph disease: Clinical, molecular, and neuropathological features

3. Analysis of SCA1, DRPLA, MJD, SCA2, and SCA6 CAG repeats in 48 portuguese ataxia families

4. A survey of spinocerebellar ataxia in South Brazil - 66 new cases with Machado-Joseph disease, SCA7, SCA8, or unidentified disease-causing mutations

5. Glutamine Repeats and Neurodegeneration

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