Extracellular vesicle-based delivery of silencing sequences for the treatment of Machado-Joseph disease/spinocerebellar ataxia type 3-Reference-Cited by-同舟云学术

Extracellular vesicle-based delivery of silencing sequences for the treatment of Machado-Joseph disease/spinocerebellar ataxia type 3

Published:2023-05 Issue:5 Volume:31 Page:1275-1292
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Rufino-Ramos David,Albuquerque Patrícia R.,Leandro Kevin,Carmona Vitor,Martins Inês M.,Fernandes Rita,Henriques Carina,Lobo Diana,Faro Rosário,Perfeito Rita,Mendonça Liliana S.,Pereira Dina,Gomes Célia M.,Nobre Rui Jorge,Pereira de Almeida Luís^ORCID

Funder

IMI

Fundacao para a Ciencia e a Tecnologia

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

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4. Autosomal dominant cerebellar ataxia type I. MRI-based volumetry of posterior fossa structures and basal ganglia in spinocerebellar ataxia types 1, 2 and 3;Klockgether;Brain.,1998

5. Ataxin-3 protein and RNA toxicity in spinocerebellar ataxia type 3: current insights and emerging therapeutic strategies;Evers;Mol. Neurobiol.,2014

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