Jove
Visualize
Contact Us
JoVE
x logofacebook logolinkedin logoyoutube logo
ABOUT JoVE
OverviewLeadershipBlogJoVE Help Center
AUTHORS
Publishing ProcessEditorial BoardScope & PoliciesPeer ReviewFAQSubmit
LIBRARIANS
TestimonialsSubscriptionsAccessResourcesLibrary Advisory BoardFAQ
RESEARCH
JoVE JournalMethods CollectionsJoVE Encyclopedia of ExperimentsArchive
EDUCATION
JoVE CoreJoVE BusinessJoVE Science EducationJoVE Lab ManualFaculty Resource CenterFaculty Site
Terms & Conditions of Use
Privacy Policy
Policies

Related Concept Videos

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...
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...
RNA Interference01:23

RNA Interference

RNA interference (RNAi) is a process in which a small non-coding RNA molecule blocks the post-transcriptional expression of a gene by binding to its messenger RNA (mRNA) and preventing the protein from being translated.
This process occurs naturally in cells, often through the activity of genomically-encoded microRNAs. Researchers can take advantage of this mechanism by introducing synthetic RNAs to deactivate specific genes for research or therapeutic purposes. For example, RNAi could be used...
RNA Interference01:23

RNA Interference

RNA interference (RNAi) is a process in which a small non-coding RNA molecule blocks the post-transcriptional expression of a gene by binding to its messenger RNA (mRNA) and preventing the protein from being translated.
This process occurs naturally in cells, often through the activity of genomically-encoded microRNAs. Researchers can take advantage of this mechanism by introducing synthetic RNAs to deactivate specific genes for research or therapeutic purposes. For example, RNAi could be used...

You might also read

Related Articles

Articles linked to this work by shared authors, journal, and citation graph.

Sort by
Same author

The <i>CTDP1</i> Founder Variant in CCFDN: Insights into Pathogenesis, Phenotypic Spectrum and Therapeutic Approaches.

International journal of molecular sciences·2026
Same author

Evaluation of a thigh-worn accelerometer for detecting leg fidgeting and estimating its energetic cost via indirect calorimetry.

Scientific reports·2026
Same author

Clinical Utility of [<sup>18</sup>F]-Fluciclovine PET/MRI for Differentiating True Progression from Treatment-Related Changes in Patients with Glioblastoma.

AJNR. American journal of neuroradiology·2025
Same author

Galectin-3 induces neurodevelopmental apical-basal polarity and regulates gyrification.

Science advances·2025
Same author

The molecular circadian clock: From fundamental mechanisms to therapeutic promise in neurological disorders.

Advanced drug delivery reviews·2025
Same author

What instruments are available to aid or evaluate personalised care delivery, from the perspectives of healthcare practitioners and service users? A narrative scoping review.

PloS one·2025

Related Experiment Video

Updated: Jul 15, 2026

Chemogenetic Regulation in Reprogrammed Stem Cell-derived Precursor Cells in Treating Neurodegenerative Diseases
09:44

Chemogenetic Regulation in Reprogrammed Stem Cell-derived Precursor Cells in Treating Neurodegenerative Diseases

Published on: May 2, 2025

RNA interference based gene therapy for neurological disease.

Aarti Jagannath1, Matthew Wood

  • 1Department of Physiology, Anatomy and Genetics, University of Oxford, South Parks Road, Oxford, UK.

Briefings in Functional Genomics & Proteomics
|May 5, 2007
PubMed
Summary

RNA interference (RNAi) shows promise for treating neurodegenerative diseases by silencing disease genes. Overcoming challenges like toxicity and immune responses is key to its therapeutic success.

More Related Videos

Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents
06:51

Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents

Published on: August 10, 2018

Lentiviral Vector Platform for the Efficient Delivery of Epigenome-editing Tools into Human Induced Pluripotent Stem Cell-derived Disease Models
13:47

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

Related Experiment Videos

Last Updated: Jul 15, 2026

Chemogenetic Regulation in Reprogrammed Stem Cell-derived Precursor Cells in Treating Neurodegenerative Diseases
09:44

Chemogenetic Regulation in Reprogrammed Stem Cell-derived Precursor Cells in Treating Neurodegenerative Diseases

Published on: May 2, 2025

Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents
06:51

Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents

Published on: August 10, 2018

Lentiviral Vector Platform for the Efficient Delivery of Epigenome-editing Tools into Human Induced Pluripotent Stem Cell-derived Disease Models
13:47

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

Area of Science:

  • Biochemistry
  • Genetics
  • Neuroscience

Background:

  • Neurodegenerative disorders lack effective small molecule drug therapies.
  • RNA interference (RNAi) offers a potential therapeutic strategy by silencing disease-causing genes.
  • RNAi has demonstrated efficacy in vitro and in animal models, with clinical trials underway.

Purpose of the Study:

  • To explore the potential of RNA interference (RNAi) as a therapeutic strategy for neurodegenerative disorders.
  • To identify and address challenges hindering the clinical application of RNAi therapy.

Main Methods:

  • Gene silencing via RNA interference (RNAi).
  • In vitro and animal model studies of RNAi efficacy.
  • Investigation of endogenous RNAi pathways, including microRNAs.

Main Results:

  • RNAi can specifically silence genes implicated in neurodegenerative diseases like spinocerebellar ataxia and Huntington's disease.
  • Therapeutic potential demonstrated in preclinical studies.
  • Challenges identified include non-specific silencing, immune responses, and pathway component saturation.

Conclusions:

  • RNAi holds significant therapeutic potential for neurodegenerative diseases.
  • Overcoming challenges such as toxicity and immune responses is crucial for safe and effective RNAi therapy.
  • Understanding endogenous RNAi mechanisms, particularly microRNAs, can optimize therapeutic strategies.