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Article

Translation of MicroRNA-Based Huntingtin-Lowering Therapies from Preclinical Studies to the Clinic.

Molecular therapy : the journal of the American Society of Gene Therapy - 4 Apr 2018

Miniarikova Jana, Evers Melvin M, Konstantinova Pavlina

Abstract excerpt

The single mutation underlying the fatal neuropathology of Huntington's disease (HD) is a CAG triplet expansion in exon 1 of the huntingtin (HTT) gene, which gives rise to a toxic mutant HTT protein. There have been a number of not yet successful therapeutic advances in the treatment of HD. The current excitement in the HD field is due to the recent development of therapies targeting the culprit of HD either at...

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Identifiers

PMID: 29503201
DOI: 10.1016/j.ymthe.2018.02.002
PMCID: PMC6079476

Genes

HTT

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 3 Sept 2026.

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