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Published on: February 9, 2016
siRNA Targeting and Treatment of Gastrointestinal Diseases
1Children's Mercy Kansas City, Kansas City, Missouri, USA.
Abstract:
RNA interference via small interfering RNA (siRNA) offers opportunities to precisely target genes that contribute to gastrointestinal (GI) pathologies, such as inflammatory bowel disease, celiac, and esophageal scarring. Delivering the siRNA to the GI tract proves challenging as the harsh environment of the intestines degrades the siRNA before it can reach its target or blocks its entry into its site of action in the cytoplasm. Additionally, the GI tract is large and disease is often localized to a specific site. This review discusses polymer and lipid-based delivery systems for protection and targeting of siRNA therapies to the GI tract to treat local disease.
Insights
Small interfering RNA (siRNA) therapies can target gastrointestinal diseases, but delivery is difficult. Polymer and lipid systems show promise for protecting and targeting siRNA to treat localized GI conditions.
Area of Science:
- Biotechnology
- Gastroenterology
- RNA Therapeutics
Background:
- Small interfering RNA (siRNA) holds potential for treating gastrointestinal (GI) pathologies like inflammatory bowel disease and celiac disease.
- Effective delivery of siRNA to the GI tract is hindered by the intestinal environment and the need for site-specific targeting.
Purpose of the Study:
- To review polymer and lipid-based delivery systems for siRNA in the GI tract.
- To discuss strategies for protecting siRNA and targeting it to specific sites for localized GI disease treatment.
Main Methods:
- Literature review of polymer and lipid-based delivery systems for siRNA.
- Analysis of challenges in GI siRNA delivery, including degradation and targeting.
Main Results:
- Polymer and lipid-based systems offer potential for protecting siRNA from degradation in the GI tract.
- These systems can be engineered for targeted delivery to specific sites within the GI tract.
Conclusions:
- Advanced delivery systems are crucial for realizing the therapeutic potential of siRNA in GI diseases.
- Targeted delivery of siRNA using polymer and lipid carriers can overcome GI tract barriers for localized treatment.
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