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siRNA therapeutics: a clinical reality
Phei Er Saw1, Er-Wei Song2,3,4
1Guangdong Provincial Key Laboratory of Malignant Tumor Epigenetics and Gene Regulation, Sun Yat-Sen Memorial Hospital, Sun Yat-Sen University, Guangzhou, 510120, China.
Science China. Life Sciences
|May 5, 2019
Summary
Small interfering RNA (siRNA) therapeutics, leveraging RNA interference (RNAi), are advancing into clinics. Key challenges remain in safe and effective siRNA delivery, despite breakthroughs like Onpattro.
Area of Science:
- Biotechnology
- Molecular Biology
- Pharmacology
Background:
- RNA interference (RNAi) discovery revolutionized gene silencing.
- Small interfering RNA (siRNA) therapeutics show immense potential for disease treatment.
- Despite progress, safe, efficient, and targeted siRNA delivery remains a significant hurdle.
Purpose of the Study:
- To review the historical progression and breakthroughs in siRNA therapeutics.
- To highlight the first FDA-approved RNAi therapeutic, Onpattro (Patisiran).
- To discuss current siRNA nanocarrier systems in clinical trials and remaining challenges.
Main Methods:
- Historical review of RNA interference (RNAi) and siRNA therapeutics.
- Analysis of key advancements and clinical applications.
- Examination of siRNA delivery systems, particularly nanocarriers.
Main Results:
- siRNA therapeutics have progressed significantly since the discovery of RNAi.
- Onpattro (Patisiran) represents a major milestone as the first FDA-approved RNAi therapeutic.
- Various siRNA nanocarriers are under investigation in clinical trials for improved delivery.
Conclusions:
- siRNA therapeutics are entering clinical practice, offering new treatment avenues.
- Effective delivery systems are crucial for the success of siRNA therapeutics.
- Overcoming remaining challenges in delivery and safety is essential for broader clinical adoption.
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