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[Gene-selective treatment approaches for Huntington's disease].

A Mühlbäck1,2, K S Lindenberg1, C Saft3

  • 1Abteilung Neurologie, Universitätsklinikum Ulm, Oberer Eselsberg 45/1, 89081, Ulm, Deutschland.

Der Nervenarzt
|March 18, 2020
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Summary

Huntington's disease (HD) is a neurodegenerative disorder affecting thousands in Germany. Innovative gene-selective therapies, including antisense oligonucleotides, show promise in slowing disease progression and mitigating symptoms when initiated early.

Keywords:
Adeno-associated virusesAntisense oligonucleotideGene therapyHuntingtinZinc finger repressor complexes

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Area of Science:

  • Neuroscience
  • Genetics
  • Pharmacology

Background:

  • Huntington's disease (HD) is a rare neurodegenerative disorder affecting approximately 8,000-14,000 individuals in Germany, with an additional 24,000 at risk.
  • The disease is characterized by relentless progression, leading to complete nursing care dependency.
  • Current medical interest is high due to emerging clinical trials offering hope for slowing HD progression.

Purpose of the Study:

  • To provide an overview of current gene-selective therapeutic approaches for Huntington's disease.
  • To highlight innovative treatments aimed at inhibiting the production of mutant huntingtin (HTT) gene products.
  • To discuss the potential of early intervention in mitigating HD clinical manifestations.

Main Methods:

  • Review of ongoing and imminent clinical studies on gene-selective therapies for HD.
  • Focus on antisense oligonucleotide (ASO) therapies, including intrathecal administration (RG6042).
  • Exploration of alternative approaches such as allele-selective ASOs, RNA-based therapies, and zinc finger repressor complexes.

Main Results:

  • A Phase III clinical trial for intrathecal antisense oligonucleotides (ASO) commenced in 2019.
  • Multiple innovative therapeutic strategies are under active investigation and development.
  • These approaches target the inhibition of mutant HTT gene product synthesis.

Conclusions:

  • Gene-selective therapies represent a promising frontier in managing Huntington's disease.
  • Early intervention with these novel treatments may significantly alter the disease's clinical course.
  • Ongoing research and clinical trials are crucial for developing effective treatments for HD.