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Related Concept Videos

Huntington Disease l: Introduction01:21

Huntington Disease l: Introduction

Huntington disease or HD is a progressive, fatal neurodegenerative disorder inherited in an autosomal dominant pattern.PathophysiologyIt is caused by expansion of the CAG trinucleotide repeat in the HTT gene on chromosome 4 (4p16.3), producing an abnormal huntingtin protein with an expanded polyglutamine tract. This misfolded protein disrupts cellular function, leading to neuronal death. Normal alleles have ≤26 repeats, 27–35 are intermediate (risk of expansion), 36–39 show reduced penetrance,...
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 inserted. The...
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 inserted. The...
Pharmacogenomics: Identification of New Drug Targets01:29

Pharmacogenomics: Identification of New Drug Targets

Advances in genomics have profoundly influenced drug discovery by increasing both the speed and accuracy of pharmaceutical development. Pharmacogenomics, which examines how genetic variation influences drug response, facilitates the identification of novel therapeutic targets and enables patient stratification for personalized treatment. These strategies contribute to improved drug efficacy, minimized adverse effects, and more efficient clinical trial design.Mapping genetic differences...
Alzheimer's Disease: Treatment01:22

Alzheimer's Disease: Treatment

Alzheimer's Disease (AD), a neurodegenerative disorder, is pathologically identified by amyloid plaques and neurofibrillary tangles composed of tau protein. AD pharmacotherapy aims to manage cognitive symptoms, delay disease progression, and treat behavioral symptoms. The treatment is primarily symptomatic and palliative, with no definitive disease-modifying therapy available. Cholinesterase inhibitors, including donepezil (Aricept), rivastigmine (Exelon), and galantamine (Razadyne), are...
Parkinson's Disease: Treatment01:24

Parkinson's Disease: Treatment

Neurodegenerative disorders, such as Parkinson's Disease (PD), involve the gradual and irreversible destruction of neurons in particular brain areas. These disorders exhibit standard features like proteinopathies, selective vulnerability of some neurons, and an interaction of intrinsic properties, genetics, and environmental influences in neural injury.
Parkinson's Disease is primarily a result of the loss of dopaminergic neurons in the substantia nigra pars compacta. The cornerstone of its...

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Related Experiment Video

Updated: May 26, 2026

Generation of Native, Untagged Huntingtin Exon1 Monomer and Fibrils Using a SUMO Fusion Strategy
11:22

Generation of Native, Untagged Huntingtin Exon1 Monomer and Fibrils Using a SUMO Fusion Strategy

Published on: June 27, 2018

Gene therapy for Huntington's disease.

Shilpa Ramaswamy1, Jeffrey H Kordower

  • 1Department of Neurological Sciences, Rush University Medical Center, Chicago, IL, USA.

Neurobiology of Disease
|January 7, 2012
PubMed
Summary

Gene therapy offers a promising avenue for treating Huntington's disease (HD), a neurodegenerative disorder. This review explores adeno-associated viral (AAV) vectors and therapeutic strategies like trophic factors and RNAi for potential HD treatments.

Area of Science:

  • Neurodegenerative diseases
  • Genetics
  • Molecular biology

Background:

  • Huntington's disease (HD) is a fatal neurodegenerative disorder with no existing cure.
  • Current therapies effective in animal models have yet to demonstrate significant benefit in human patients.
  • Gene therapy presents a potential therapeutic strategy for managing HD.

Purpose of the Study:

  • To review the application of adeno-associated viral (AAV) vectors for gene therapy delivery in the brain.
  • To discuss therapeutic approaches, including trophic factors and RNA interference (RNAi), for Huntington's disease.
  • To evaluate the potential of these gene-based therapies for treating HD.

Main Methods:

  • Review of scientific literature on gene therapy for Huntington's disease.

More Related Videos

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

Efficient and Scalable Production of Full-length Human Huntingtin Variants in Mammalian Cells using a Transient Expression System
10:52

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

Published on: December 10, 2021

Related Experiment Videos

Last Updated: May 26, 2026

Generation of Native, Untagged Huntingtin Exon1 Monomer and Fibrils Using a SUMO Fusion Strategy
11:22

Generation of Native, Untagged Huntingtin Exon1 Monomer and Fibrils Using a SUMO Fusion Strategy

Published on: June 27, 2018

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

Efficient and Scalable Production of Full-length Human Huntingtin Variants in Mammalian Cells using a Transient Expression System
10:52

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

Published on: December 10, 2021

  • Examination of adeno-associated viral (AAV) vector systems for brain delivery.
  • Analysis of therapeutic compounds such as trophic factors and RNAi targeting mutant huntingtin (mHtt) protein.

    • Main Results:

    • Adeno-associated viral (AAV) vectors are a viable method for long-term, widespread gene therapy delivery to the brain.
    • Trophic factors show potential in protecting neurons from degeneration in HD models.
    • RNAi-based strategies can effectively reduce the expression of mutant huntingtin (mHtt) protein.

    Conclusions:

    • Gene therapy, particularly using AAV vectors, holds significant promise for treating Huntington's disease.
    • Combining AAV delivery with neuroprotective trophic factors or RNAi offers a multi-faceted therapeutic approach.
    • Further research and clinical translation of these gene-based strategies are warranted for HD treatment.