Gentherapie der Huntington-Krankheit

Research output: Contribution to journalReview articleContributedpeer-review

Contributors

Abstract

Englisch: Being one of the most common genetic neurodegenerative disease, Huntington's disease has been a model disease - also for gene therapy. Among the various options, the development of antisense oligonucleotides is the most advanced. Further options at the RNA level include micro-RNAs and modulators of RNA processing (splicing), at the DNA level zinc finger proteins. Several products are in clinical trials. These differ in their mode of application and in the extent of systemic availability. Another important difference between therapeutic strategies could be whether all forms of the huntingtin protein are targeted in the same extent, or whether a therapy preferentially targets particular toxic forms such as the exon1 protein. The results of the recently terminated GENERATION HD1 trial were somewhat sobering, most likely due to the side effect-related hydrocephalus. Therefore they represent just one step towards the development of an effective gene therapy against Huntington's disease.

Translated title of the contribution
Gene Therapy for Huntington Disease

Details

Original languageGerman
Pages (from-to)141-146
Number of pages6
JournalFortschritte der Neurologie, Psychiatrie : FDN
Volume91
Issue number4
Publication statusPublished - Apr 2023
Peer-reviewedYes

External IDs

PubMedCentral PMC10089766
Scopus 85152298058
ORCID /0000-0002-2387-526X/work/150328944

Keywords

Keywords

  • Humans, Huntington Disease/genetics, Neurodegenerative Diseases, Genetic Therapy/methods, Oligonucleotides, Antisense/genetics, Huntingtin Protein/genetics