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Published on: August 17, 2022
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Oral nucleic acid therapy using multicompartmental delivery systems.
Husain Attarwala1, Murui Han1, Jonghan Kim1
1Department of Pharmaceutical Sciences, School of Pharmacy, Bouve College of Health Sciences, Northeastern University, Boston, MA, USA.
Summary
Developing novel oral delivery systems for nucleic acid therapeutics is crucial for treating genetic diseases. A multicompartmental system effectively protected nucleic acids in the gastrointestinal tract, enabling gene therapy for colitis in mice.
Area of Science:
- Nanotechnology
- Biotechnology
- Drug Delivery
Background:
- Nucleic acid therapeutics offer potential for treating genetic diseases by modulating gene expression.
- Clinical advancement is hindered by the lack of safe and effective delivery strategies, especially for oral administration.
- The gastrointestinal tract presents significant barriers, including enzymatic degradation and cellular uptake challenges, for oral nucleic acid delivery.
Purpose of the Study:
- To review multicompartmental systems for overcoming extracellular and intracellular barriers to oral nucleic acid delivery.
- To present a novel nanoparticles-in-microsphere oral system for in vivo gene and small interfering RNA delivery.
- To evaluate the efficacy of this system in a mouse model of intestinal bowel disease.
Main Methods:
- Discussion of multicompartmental systems designed to protect nucleic acids within the gastrointestinal tract.
- Development and testing of a nanoparticles-in-microsphere oral delivery system.
- In vivo assessment of gene and small interfering RNA delivery for treating colitis in mice.
Main Results:
- The developed multicompartmental system demonstrated protection of nucleic acids during transit through the gastrointestinal tract.
- Efficient transgene expression and gene silencing were achieved following oral administration of the system.
- The oral delivery system exhibited favorable anti-inflammatory effects in a mouse model of intestinal bowel disease.
Conclusions:
- Multicompartmental systems represent a promising strategy for overcoming the challenges of oral nucleic acid delivery.
- The tested nanoparticles-in-microsphere system shows potential for therapeutic applications, including the treatment of inflammatory bowel disease.
- Further development of such systems could significantly advance the clinical utility of nucleic acid-based therapeutics.
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