Related Experiment Video

Updated: May 18, 2026

Intrathecal Delivery of Antisense Oligonucleotides in the Rat Central Nervous System
07:47

Intrathecal Delivery of Antisense Oligonucleotides in the Rat Central Nervous System

Published on: October 29, 2019

RNAi-based therapies for Huntington's disease: delivery challenges and opportunities

Neelima Mantha1, Sudip K Das, Nandita G Das

  • 1Department of Pharmaceutica Sciences, College of Pharmacy & Health Sciences, Butler University, Indianapolis, IN 46208, USA.

Therapeutic Delivery
|October 6, 2012
PubMed

Insights

Huntington's disease (HD) is a neurodegenerative disorder with no cure. RNA interference (RNAi) offers potential treatment by targeting the Huntingtin gene (HTT), but drug delivery challenges remain a key focus for novel therapeutic approaches.

Area of Science:

  • Neurodegenerative diseases
  • Genetics
  • Molecular biology

Background:

  • Huntington's disease (HD) is a fatal neurodegenerative disorder.
  • It is caused by a mutation in the Huntingtin gene (HTT), leading to an abnormal Huntingtin protein.
  • Current treatments only manage symptoms, not disease progression.

Purpose of the Study:

  • To explore RNA interference (RNAi) as a therapeutic strategy for Huntington's disease.
  • To discuss the challenges associated with using RNAi for HD treatment.
  • To present novel approaches for overcoming drug delivery obstacles in RNAi-based HD therapy.

Main Methods:

  • Review of RNAi technology principles and applications in neurodegenerative disease models.
  • Analysis of existing challenges in delivering RNAi therapeutics to the central nervous system.
  • Exploration of innovative drug delivery systems and strategies for HD.

Main Results:

  • RNAi holds significant promise for targeting the root cause of HD by reducing mutant Huntingtin protein levels.
  • Significant hurdles exist in achieving effective and safe delivery of RNAi agents to the brain.
  • Emerging delivery technologies show potential for improved therapeutic efficacy.

Conclusions:

  • RNAi-based therapies represent a promising avenue for treating Huntington's disease.
  • Overcoming drug delivery challenges is critical for the clinical success of RNAi in HD.
  • Continued research into novel delivery methods is essential for advancing HD therapeutics.

More Related Videos

Lumbar Intrathecal Injection of SOD1-ASOs for Precise CNS Targeting and Predictive Efficacy in Human SOD1-G93A ALS Mice04:41

Lumbar Intrathecal Injection of SOD1-ASOs for Precise CNS Targeting and Predictive Efficacy in Human SOD1-G93A ALS Mice

Published on: February 24, 2026

Delivery of Therapeutic Agents Through Intracerebroventricular (ICV) and Intravenous (IV) Injection in Mice05:55

Delivery of Therapeutic Agents Through Intracerebroventricular (ICV) and Intravenous (IV) Injection in Mice

Published on: October 3, 2011

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,...
Experimental RNAi02:15

Experimental RNAi

RNA interference (RNAi) is a cellular mechanism that inhibits gene expression by suppressing its transcription or activating the RNA degradation process. The mechanism was discovered by Andrew Fire and Craig Mello in 1998 in plants. Today, it is observed in almost all eukaryotes, including protozoa, flies, nematodes, insects, parasites, and mammals. This precise cellular mechanism of gene silencing has been developed into a technique that provides an efficient way to identify and determine 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...
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...
siRNA - Small Interfering RNAs02:30

siRNA - Small Interfering RNAs

Small interfering RNAs, or siRNAs, are short regulatory RNA molecules that can silence genes post-transcriptionally, as well as the transcriptional level in some cases. siRNAs are important for protecting cells against viral infections and silencing transposable genetic elements.
In the cytoplasm, siRNA is processed from a double-stranded RNA, which comes from either endogenous DNA transcription or exogenous sources like a virus. This double-stranded RNA is then cleaved by the ATP-dependent...
Microorganisms in Medicine and Therapeutics01:29

Microorganisms in Medicine and Therapeutics

Microorganisms play a fundamental role in vaccine development, gene therapy, and therapeutic production. Their biological properties are harnessed to advance medicine and public health. Beyond immunization, microorganisms contribute to gut health, antibiotic synthesis, and genetic disease treatment.Live Attenuated and Inactivated VaccinesLive attenuated vaccines, such as the measles, mumps, and rubella (MMR) vaccine, utilize weakened forms of pathogens to closely resemble natural infections.