Repeated Mesenchymal Stromal Cell Treatment Sustainably Alleviates Machado-Joseph Disease

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...

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Detaylı Bibliyografya
Yayımlandı:Mol Ther
Asıl Yazarlar: Oliveira Miranda, Catarina, Marcelo, Adriana, Silva, Teresa Pereira, Barata, João, Vasconcelos-Ferreira, Ana, Pereira, Dina, Nóbrega, Clévio, Duarte, Sónia, Barros, Inês, Alves, Joana, Sereno, José, Petrella, Lorena Itatí, Castelhano, João, Paiva, Vitor Hugo, Rodrigues-Santos, Paulo, Alves, Vera, Nunes-Correia, Isabel, Nobre, Rui Jorge, Gomes, Célia, Castelo-Branco, Miguel, Pereira de Almeida, Luís
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: American Society of Gene & Cell Therapy 2018
Konular:
Original Article
Online Erişim:https://ncbi.nlm.nih.gov/pmc/articles/PMC6127516/
https://ncbi.nlm.nih.gov/pubmed/30087083
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2018.07.007
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