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Erschienen in: Current Neurology and Neuroscience Reports 3/2021

01.03.2021 | Movement Disorders (T. Simuni, Section Editor)

Huntington’s Disease: New Frontiers in Therapeutics

verfasst von: Ling Pan, Andrew Feigin

Erschienen in: Current Neurology and Neuroscience Reports | Ausgabe 3/2021

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Abstract

Purpose of Review

This article describes and discusses new potential disease-modifying therapies for Huntington’s disease that are currently in human clinical trials as well as promising new therapies in preclinical development.

Recent Findings

Multiple potential disease-modifying therapeutics for HD are in active development, including direct DNA/gene therapies, RNA modulation, and therapies targeted at aberrant downstream pathways.

Summary

The etiology of Huntington’s disease (HD) is well-known as an abnormally expanded trinucleotide repeat within the huntingtin gene. However, the pathogenesis downstream of the mutant huntingtin gene is complex, involving multiple toxic pathways, including abnormal protein fragmentation and neuroinflammation. The current treatment of HD focuses largely on symptomatic management. This article discusses new, potential disease-modifying therapies that are currently in human clinical trials and preclinical development.
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Metadaten
Titel
Huntington’s Disease: New Frontiers in Therapeutics
verfasst von
Ling Pan
Andrew Feigin
Publikationsdatum
01.03.2021
Verlag
Springer US
Erschienen in
Current Neurology and Neuroscience Reports / Ausgabe 3/2021
Print ISSN: 1528-4042
Elektronische ISSN: 1534-6293
DOI
https://doi.org/10.1007/s11910-021-01093-3

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