Open AccessNon-invasive and allele-specific silencing therapy for Polyglutamine disorders: the case of Machado-Joseph disease/Spinocerebellar ataxia type 3
Author(s) -
Rui Jorge Nobre,
Joana Saraiva,
Susana Paixão,
Cellia Fusco,
Magda M. Santana,
Catarina Miranda,
Lorena Petrella,
José Sereno,
João Castelhano,
Miguel CasteloBranco,
Miguel SenaEsteves,
Luís Pereira de Almeida
Publication year - 2019
Publication title -
frontiers in cellular neuroscience
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.877
H-Index - 86
ISSN - 1662-5102
DOI - 10.3389/conf.fncel.2019.01.00007
Subject(s) - spinocerebellar ataxia , machado–joseph disease , gene silencing , trinucleotide repeat expansion , biology , rna interference , viral vector , ataxia , genetics , virology , allele , neuroscience , rna , gene , recombinant dna