Related Experiment Video

Updated: Nov 25, 2025

Fractionation for Resolution of Soluble and Insoluble Huntingtin Species
07:08

Fractionation for Resolution of Soluble and Insoluble Huntingtin Species

Published on: February 27, 2018

9.9K

Strategies for mutant gene expression silencing in Huntington’s disease therapy

Agnieszka Fiszer1, Bartosz Nowak2

  • 1Department of Medical Biotechnology, Institute of Bioorganic Chemistry Polish Academy of Sciences, Poznan. agnieszka.fiszer@ibch.poznan.pl.

Postepy Biochemii
|December 15, 2020
PubMed

Insights

Huntington's disease (HD) treatments focus on gene silencing. This review highlights antisense oligonucleotides (ASO), RNA interference (RNAi), and CRISPR-Cas9 as key strategies to combat neurodegeneration in HD patients.

Area of Science:

  • Genetics
  • Neuroscience
  • Molecular Biology

Background:

  • Huntington's disease (HD) is a progressive neurodegenerative disorder.
  • It is caused by an expanded CAG repeat in the HTT gene, leading to mutant huntingtin protein.
  • Current life expectancy after symptom onset is 15-20 years, highlighting the need for effective treatments.

Purpose of the Study:

  • To review current therapeutic strategies for Huntington's disease.
  • To focus on gene expression silencing approaches.
  • To discuss ongoing clinical trials and pharmacological agents.

Main Methods:

  • Review of current therapeutic strategies for HD.
  • Emphasis on gene silencing mechanisms.
  • Discussion of antisense oligonucleotides (ASO), RNA interference (RNAi), and CRISPR-Cas9 technologies.

Main Results:

  • Various gene silencing strategies are under investigation for HD.
  • Antisense oligonucleotides (ASO), RNA interference (RNAi), and CRISPR-Cas9 are prominent approaches.
  • Ongoing clinical trials are evaluating these and other pharmacological agents.

Conclusions:

  • Gene silencing offers promising therapeutic avenues for Huntington's disease.
  • ASO, RNAi, and CRISPR-Cas9 represent key technologies in HD treatment development.
  • Further research and clinical trials are crucial for advancing HD therapies.

More Related Videos

Lentiviral Vector Platform for the Efficient Delivery of Epigenome-editing Tools into Human Induced Pluripotent Stem Cell-derived Disease Models13:47

Lentiviral Vector Platform for the Efficient Delivery of Epigenome-editing Tools into Human Induced Pluripotent Stem Cell-derived Disease Models

Published on: March 29, 2019

10.0K
Efficient and Scalable Production of Full-length Human Huntingtin Variants in Mammalian Cells using a Transient Expression System10:52

Efficient and Scalable Production of Full-length Human Huntingtin Variants in Mammalian Cells using a Transient Expression System

Published on: December 10, 2021

2.8K

Related Concept Videos

In-vitro Mutagenesis01:16

In-vitro Mutagenesis

To learn more about the function of a gene, researchers can observe what happens when the gene is inactivated or “knocked out,” by creating genetically engineered knockout animals. Knockout mice have been particularly useful as models for human diseases such as cancer, Parkinson’s disease, and diabetes.
15.7K
Gene Therapy00:59

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
26.9K