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Delivery of Therapeutic siRNA to the CNS Using Cationic and Anionic Liposomes
Published on: July 23, 2016
Liposome-siRNA-peptide complexes cross the blood-brain barrier and significantly decrease PrP on neuronal cells and
Bruce Pulford1, Natalia Reim, Aimee Bell
1Department of Microbiology, College of Veterinary Medicine and Biomedical Sciences, Colorado State University, Fort Collins, Colorado, United States of America.
Plos One
|June 19, 2010
Summary
A novel liposome-siRNA-peptide complex (LSPC) effectively delivers prion-targeting siRNA to neurons. This new system suppresses prion protein expression and formation in vitro and in vivo, offering a promising therapeutic strategy for prion diseases.
Area of Science:
- Neuroscience
- Molecular Biology
- Drug Delivery Systems
Background:
- Prion diseases are linked to host PrP(C) expression levels.
- RNA interference is a potential therapeutic strategy for prion diseases.
- Current lentivirus delivery systems show limited in vivo efficacy.
Purpose of the Study:
- To develop a novel siRNA delivery system for prion diseases.
- To enhance siRNA delivery specifically to neurons.
- To improve therapeutic efficacy for prion neuropathology.
Main Methods:
- Developed a liposome-siRNA-peptide complex (LSPC).
- The peptide component targets acetylcholine receptors (AchRs) on neurons.
- Cationic liposomes protect siRNA-peptide complexes from serum degradation.
Main Results:
- LSPCs delivered PrP siRNA specifically to AchR-expressing cells.
- PrP(C) expression was suppressed and PrP(RES) formation eliminated in vitro.
- Intravenous LSPC injection in mice led to brain-wide delivery to neurons, resisting serum degradation.
Conclusions:
- LSPCs are effective vehicles for neuronal siRNA delivery.
- This system shows potential for treating prion and other neuropathological diseases.
- LSPCs offer a promising approach for targeted gene silencing in the brain.
