Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2023 Oct;53(10):e14039.
doi: 10.1111/eci.14039. Epub 2023 Jun 13.

Small interference (RNAi) technique: Exploring its clinical applications, benefits and limitations

Moqbel Ali Moqbel Redhwan  1   2 , Hariprasad M G  1   2 , Suman Samaddar  3 , Sumaia Abdulbari Ahmed Ali Hard  2   4 , Vidyabhushan Yadav  1 , Apurbo Mukherjee  1 , Rahul Kumar  1
Affiliations
Review

Small interference (RNAi) technique: Exploring its clinical applications, benefits and limitations

Moqbel Ali Moqbel Redhwan et al. Eur J Clin Invest. 2023 Oct.

Abstract

Background: Small interference RNA (siRNA) has emerged as the most desired method for researchers and clinicians who wish to silence a specific gene of interest and has been extensively developed as a therapeutic agent. This review points to collecting all clinical trials on siRNA and understanding its benefits, pharmacokinetics and safety by reading articles published in the last 5 years.

Materials and methods: Searching in the PubMed database using 'siRNA' and 'in vivo' with limits to articles published in the previous 5 years, article type 'clinical trials' and language 'English' to acquire papers on in vivo studies on siRNA approaches. Features of siRNA clinical trials registered at https://clinicaltrials.gov/ were analysed.

Results: So far, 55 clinical studies have been published on siRNA. Many published clinical trials on siRNA showed tolerability, safety and effectiveness in treating cancers like breast, lung, colon, and other organs and other diseases like viral infections and hereditary diseases. Many different routes of administration can silence many genes at the same time. Limitations and uncertainties associated with siRNA treatment include the effectiveness of cellular uptake, precise targeting of the intended tissue or cell and prompt elimination from the body.

Conclusions: The siRNA or RNAi method will be one of the most critical and influential techniques to fight against many different diseases. Although the RNAi approach has certain advantages, it also has limitations concerning clinical applications. Overcoming these limitations remains a daunting challenge.

Keywords: RNA interference; clinical application; small interference RNA; treatment.

PubMed Disclaimer

References

REFERENCES

    1. Aagaard L, Rossi JJ. RNAi therapeutics: principles, prospects, and challenges. Adv Drug Deliv Rev. 2007;59:75-86.
    1. Braasch DA, Jensen S, Liu Y, et al. RNA interference in mammalian cells by chemically-modified RNA. Biochemistry. 2033;42:7967-7975.
    1. Ralph GS, Radcliffe PA, Day DM, et al. Silencing mutant SOD1 using RNAi protects against neurodegeneration and extends survival in an ALS model. Nat Med. 2005;11:429-433.
    1. Ge Q, Filip L, Bai A, Nguyen T, Eisen HN, Chen J. Inhibition of influenza virus production in virus-infected mice by RNA interference. Proc Natl Acad Sci U S A. 2004;101:8676-8681.
    1. Qin ZH, Wang J, Gu ZL. Development of novel therapies for Huntington's disease: hope and challenge. Acta Pharmacol Sin. 2005;26:129-142.

Substances

LinkOut - more resources