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Article

Intravenous administration of brain-targeted stable nucleic acid lipid particles alleviates Machado-Joseph disease neurological phenotype.

Biomaterials - 1 Mar 2016

Conceição Mariana, Mendonça Liliana, Nóbrega Clévio, Gomes Célia, Costa Pedro, Hirai Hirokazu, Moreira João Nuno, Lima Maria C, Manjunath N, Pereira de Almeida Luís

Abstract excerpt

Others and we showed that RNA interference holds great promise for the treatment of dominantly inherited neurodegenerative disorders such as Machado-Joseph disease (MJD), for which there is no available treatment. However, successful experiments involved intracranial administration of viral vectors and there is a need for a safer and less invasive procedure. In this work, we successfully generated stable nucleic...

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Identifiers

PMID: 26757259
DOI: 10.1016/j.biomaterials.2015.12.021

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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