Ontology highlight
ABSTRACT:
SUBMITTER: Oliveira Miranda C
PROVIDER: S-EPMC6127516 | biostudies-literature | 2018 Sep
REPOSITORIES: biostudies-literature
Oliveira Miranda Catarina C Marcelo Adriana A Silva Teresa Pereira TP Barata João J Vasconcelos-Ferreira Ana A Pereira Dina D Nóbrega Clévio C Duarte Sónia S Barros Inês I Alves Joana J Sereno José J Petrella Lorena Itatí LI Castelhano João J Paiva Vitor Hugo VH Rodrigues-Santos Paulo P Alves Vera V Nunes-Correia Isabel I Nobre Rui Jorge RJ Gomes Célia C Castelo-Branco Miguel M Pereira de Almeida Luís L
Molecular therapy : the journal of the American Society of Gene Therapy 20180712 9
Machado-Joseph disease (MJD) or spinocerebellar ataxia type 3, the most common dominant spinocerebellar ataxia (SCA) worldwide, is caused by over-repetition of a CAG repeat in the ATXN3/MJD1 gene, which translates into a polyglutamine tract within the ataxin-3 protein. There is no treatment for this fatal disorder. Despite evidence of the safety and efficacy of mesenchymal stromal cells (MSCs) in delaying SCA disease progression in exploratory clinical trials, unanticipated regression of patient ...[more]