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Gene Therapy

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...

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Updated: Jun 6, 2025

Delivery of Therapeutic siRNA to the CNS Using Cationic and Anionic Liposomes

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Published on: July 23, 2016

Delivering therapeutic RNA into the brain using extracellular vesicles.

Paniz Shirmast^1,2, Mahdi Abedinzadeh Shahri¹, Austin Brent^1,2

¹Institute for Biomedicine and Glycomics, Griffith University, Brisbane, QLD, Australia.

Molecular Therapy. Nucleic Acids

|December 2, 2024

Summary

Extracellular vesicles (EVs) offer a promising solution for delivering RNA therapeutics to the brain, overcoming the blood-brain barrier challenge. These natural nanoparticles protect RNA from degradation, advancing neurological disorder treatments.

Keywords:

MT: Delivery Strategies RNA interference blood-brain barrier extracellular vesicles lipid nanoparticles neurodegenerative disease therapeutic RNA

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Published on: November 8, 2024

Area of Science:

RNA therapeutics
Neuroscience
Nanomedicine

Background:

RNA-based therapies show great promise but face delivery challenges, especially across the blood-brain barrier.
Conventional delivery methods like lipid nanoparticles are often excluded by the blood-brain barrier and RNA is prone to degradation.
Extracellular vesicles (EVs) are natural nanoparticles that protect cargo and have emerged as a potential solution for RNA delivery.

Purpose of the Study:

To review current RNA delivery strategies for brain applications.
To highlight the challenges associated with conventional RNA delivery methods.
To emphasize the potential of EV-mediated RNA delivery for neurological disorders.

Main Methods:

Literature review of RNA delivery strategies.
Analysis of challenges in crossing the blood-brain barrier.
Evaluation of extracellular vesicles as RNA carriers.

Main Results:

Conventional RNA delivery methods face significant hurdles in brain targeting due to the blood-brain barrier.
Extracellular vesicles demonstrate inherent protection for RNA cargo and efficient cellular uptake.
EVs show promise for safe and effective delivery of RNA therapeutics to the brain.

Conclusions:

Extracellular vesicles represent a promising platform for advancing RNA-based therapeutics for neurological disorders.
Further research into RNA/EV brain delivery strategies is crucial to realize their therapeutic potential.
EV-mediated delivery offers a viable path to overcome current limitations in treating brain diseases with RNA.