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siRNA - Small Interfering RNAs

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...

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Engineered nanoparticles for systemic siRNA delivery to malignant brain tumours.

Johan Karlsson¹, Yuan Rui², Kristen L Kozielski²

¹Department of Biomedical Engineering and the Translational Tissue Engineering Center, Johns Hopkins University School of Medicine, Baltimore, MD 21231, USA. green@jhu.edu and Institute for Nanobiotechnology, Johns Hopkins University, Baltimore, MD 21218, USA. searson@jhu.edu and Department of Materials Science and Engineering, Johns Hopkins University, Baltimore, MD 21218, USA.

|October 16, 2019

Summary

Engineered nanoparticles effectively deliver siRNA across the blood-brain barrier (BBB) for brain cancer treatment. This biodegradable nanocarrier enables gene silencing in glioblastoma cells, opening new avenues for neurological disease therapies.

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Preparation of Neutrally-charged, pH-responsive Polymeric Nanoparticles for Cytosolic siRNA Delivery

Preparation of Neutrally-charged, pH-responsive Polymeric Nanoparticles for Cytosolic siRNA Delivery

Published on: May 2, 2019

Area of Science:

Biomedical Engineering
Nanotechnology
Neuroscience

Background:

Effective siRNA delivery across the blood-brain barrier (BBB) is crucial for treating brain cancers like glioblastoma.
Current delivery systems face challenges in crossing this biological barrier.
Novel nanomedicines are needed to transport therapeutic oligonucleotides to the brain.

Purpose of the Study:

To engineer bioreducible nanoparticles for systemic siRNA delivery to glioblastoma cells.
To evaluate and optimize nanoparticle performance in crossing the BBB using a novel in vitro model.
To demonstrate the therapeutic potential of these nanoparticles in an orthotopic mouse tumor model.

Main Methods:

Development of bioreducible nanoparticles for siRNA encapsulation.
Utilized a biomimetic in vitro model of the brain microvascular endothelium to assess BBB penetration.
Transmission electron microscopy for visualizing nanoparticle transport mechanism.
In vivo studies in an orthotopic mouse glioblastoma model for efficacy and safety assessment.

Main Results:

Engineered nanoparticles successfully crossed the in vitro BBB model via a vesicular mechanism.
Optimized nanoparticles demonstrated safe delivery across the BBB in vivo.
Nanoparticles were internalized by human brain cancer cells, releasing siRNA into the cytosol.
Achieved significant gene silencing both in vitro and in vivo in the tumor model.

Conclusions:

The study presents a novel bioreducible nanocarrier for systemic siRNA delivery to the brain.
The developed in vitro BBB model is effective for evaluating and engineering nanomedicines for brain delivery.
This biodegradable nanocarrier shows promise for treating glioma and other neurological diseases through gene silencing.