Related Experiment Video
Updated: Aug 10, 2026
07:08
Fractionation for Resolution of Soluble and Insoluble Huntingtin Species
Published on: February 27, 2018
Nucleic Acid Therapeutics in Huntington's Disease
1Department of Biotechnology, National Institute of Pharmaceutical Education and Research (NIPER), Sector 67, S.A.S. Nagar, Punjab 160062, India.
Recent Patents on Biotechnology
|February 13, 2019
Summary
Nucleic acid therapeutics offer a selective approach to downregulate mutant huntingtin expression, reducing protein aggregation in Huntington's disease (HD). Antisense oligonucleotides and RNA interference show promise in preclinical and early clinical studies for HD treatment.
Area of Science:
- Neuroscience
- Genetics
- Molecular Biology
Background:
- Protein misfolding and aggregation are central to neurodegenerative diseases like Huntington's disease (HD).
- Mutant huntingtin protein aggregates form neuronal inclusions, a hallmark of HD pathology.
- Conventional protein stabilizers have limited success in inhibiting misfolding and aggregation.
Purpose of the Study:
- To review the emerging role of nucleic acid therapeutics in targeting mutant huntingtin.
- To explore strategies for using nucleic acids to downregulate aberrant gene expression in HD.
- To assess the potential of nucleic acid-based therapies for Huntington's disease.
Main Methods:
- Review of patent databases (EPO, USPTO, WIPO, Google Patents) for nucleic acid therapeutics.
- Analysis of antisense oligonucleotides (ASOs), short interfering RNA (siRNA), and aptamers targeting mutant huntingtin.
- Evaluation of studies in cellular and animal models of HD, including limited clinical trials.
Main Results:
- RNA interference (RNAi) and ASOs effectively reduce mutant huntingtin mRNA and protein levels.
- Nucleic acid sequences target CAG repeats and single nucleotide polymorphisms associated with HD.
- Early clinical trials show huntingtin protein downregulation with ASOs; RNA aptamers show promise in yeast models.
- Novel drug delivery methods are being developed to overcome the blood-brain barrier.
Conclusions:
- Nucleic acid therapeutics offer high selectivity and specificity for targeting mutant huntingtin.
- Advanced delivery techniques enhance the potential of these agents for treating HD.
- Nucleic acid-based strategies represent a promising avenue for a curative approach to Huntington's disease.
Keywords:
Antisense oligonucleotidesRNA silencingaptamersgene therapyprotein aggregationproteostasis networksiRNA.More Related Videos
10:52Efficient and Scalable Production of Full-length Human Huntingtin Variants in Mammalian Cells using a Transient Expression System
Published on: December 10, 2021
04:41Lumbar Intrathecal Injection of SOD1-ASOs for Precise CNS Targeting and Predictive Efficacy in Human SOD1-G93A ALS Mice
Published on: February 24, 2026
Related Concept Videos
02:30siRNA - 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...
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...
01:24Parkinson'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...
Parkinson's Disease is primarily a result of the loss of dopaminergic neurons in the substantia nigra pars compacta. The cornerstone of its...
01:22Alzheimer'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...
01:29Pharmacogenomics: 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...
01:22Antiviral Nucleoside Inhibitors
Antiviral Nucleoside InhibitorsAntiviral nucleoside inhibitors are structural analogs of natural nucleosides that interfere with viral DNA or RNA synthesis. These compounds selectively target viral polymerases due to their resemblance to host nucleosides, thereby disrupting viral genome replication.Mechanism of Acyclovir ActionAcyclovir is a guanosine analog with a three-carbon acyclic side chain. It selectively targets herpes simplex virus type 1 (HSV-1), herpes simplex virus type 2 (HSV-2),...
01:21Huntington 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,...