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Related Concept Videos

Gene Therapy00:59

Gene Therapy

25.4K
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...
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siRNA - Small Interfering RNAs02:30

siRNA - Small Interfering RNAs

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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...
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Related Experiment Video

Updated: Jun 27, 2025

Delivery of Therapeutic siRNA to the CNS Using Cationic and Anionic Liposomes
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Nanoparticle-based Gene Therapy for Neurodegenerative Disorders.

Nelofer Ereej1, Huma Hameed1, Mahtab Ahmad Khan1,2

  • 1Faculty of Pharmaceutical Sciences, University of Central Punjab, Lahore 54000, Pakistan.

Mini Reviews in Medicinal Chemistry
|April 27, 2024
PubMed
Summary

Nanoparticle gene therapy offers a promising approach for neurological disorders by enabling targeted gene delivery across the blood-brain barrier. Further research is needed to ensure safety and efficacy in clinical applications.

Keywords:
Nanoparticleblood-brain barrier.gene therapynanomedicinenanoparticlesneurological disorderstargeted

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Area of Science:

  • Neuroscience
  • Biotechnology
  • Materials Science

Background:

  • Neurological disorders pose significant challenges due to brain complexity and genetic intervention difficulties.
  • Current treatments for neurological disorders are limited in efficacy and scope.

Purpose of the Study:

  • To review nanoparticle-mediated gene therapy for neurological disorders.
  • To highlight advancements, challenges, and future directions in this therapeutic area.

Main Methods:

  • Exploration of nanoparticle synthesis and gene conjugation techniques.
  • Review of nanoparticle properties relevant to gene delivery, including targeting, stability, and immunogenicity.
  • Analysis of the potential to overcome blood-brain barrier restrictions.

Main Results:

  • Nanoparticles offer precise targeting, enhanced stability, and potential to bypass the blood-brain barrier for gene delivery.
  • Various nanoparticle materials and designs allow for specific tissue targeting and improved circulation stability.
  • Low immunogenicity is a key advantage of these nanoparticle systems.

Conclusions:

  • Nanoparticle-based gene therapy is a promising strategy for treating neurological disorders.
  • Further clinical trials and nanosafety research are essential to validate therapeutic applications.
  • Continued research is crucial to unlock the full potential of this innovative approach for neurological health.