Logo Medical Science Monitor

Call: +1.631.470.9640
Mon - Fri 10:00 am - 02:00 pm EST

Contact Us

Logo Medical Science Monitor Logo Medical Science Monitor Logo Medical Science Monitor

10 April 2006

RNAi: A novel antisense technology and its therapeutic potential.

Anne Dallas, Alexander V. Vlassov

Med Sci Monit 2006; 12(4): RA67-74 :: ID: 448934


Antisense oligonucleotide agents induce the inhibition of target gene expressionin a sequence-specific manner by exploiting the ability of oligonucleotides to bind to target RNAs viaWatson-Crick hybridization. Once bound, the antisense agent either disables or induces the degradationof the target RNA. This technology may be used for therapeutic purposes, functional genomics, and targetvalidation. There are three major categories of gene-silencing molecules: (1) antisense oligonucleotidederivatives that, depending on their type, recruit RNase H to cleave the target mRNA or inhibit translationby steric hindrance; (2) ribozymes and deoxyribozymes - catalytically active oligonucleotides that causeRNA cleavage; (3) small interfering double-stranded RNA molecules that induce RNA degradation througha natural gene-silencing pathway called RNA interference (RNAi). RNAi is the latest addition to the familyof antisense technologies and has rapidly become the most widely used approach for gene knockdown becauseof its potency. In this mini-review, we introduce the RNAi effect, briefly compare it with existing antisensetechnologies, and discuss its therapeutic potential, focusing on recent animal studies and ongoing clinicaltrials. RNAi may provide new therapeutics for treating viral infections, neurodegenerative diseases,septic shock, macular degeneration, cancer, and other illnesses, although in vivo delivery of small interferingRNAs remains a significant obstacle.

Keywords: Neoplasms - therapy, Neurodegenerative Diseases - therapy, Oligonucleotides, Antisense - genetics, RNA Interference, RNA, Small Interfering - therapeutic use, Virus Diseases - therapy



01 August 2022 : Editorial  

Editorial: Long-Term Effects of Symptomatic and Asymptomatic SARS-CoV-2 Infection in Children and the Changing Pathogenesis of Common Childhood Viruses Driven by the COVID-19 Pandemic

Dinah V. Parums
Science Editor, Medical Science Monitor, International Scientific Information, Inc., Melville, NY, USA

DOI: 10.12659/MSM.937927

Med Sci Monit 2022; 28:e937927

In Press

08 Aug 2022 : Clinical Research  

A 3-Year Clinical Evaluation of Endodontically Treated Posterior Teeth Restored with Resin Nanoceramic Comp...

Med Sci Monit In Press; DOI: 10.12659/MSM.937331  

08 Aug 2022 : Clinical Research  

Hemoglobin/Red Blood Cell Distribution Width Ratio in Peripheral Blood Is Positively Associated with Progno...

Med Sci Monit In Press; DOI: 10.12659/MSM.937146  

05 Aug 2022 : Clinical Research  

A Retrospective Study of 98 Elderly Patients with High-Risk Lateral Femoral Wall Intertrochanteric Hip Frac...

Med Sci Monit In Press; DOI: 10.12659/MSM.936923  

04 Aug 2022 : Clinical Research  

Radiological Assessment of Prevalance and Quality of Periapical Status of Endodontic Treatments

Med Sci Monit In Press; DOI: 10.12659/MSM.936569  

Most Viewed Current Articles

30 Dec 2021 : Clinical Research  

Retrospective Study of Outcomes and Hospitalization Rates of Patients in Italy with a Confirmed Diagnosis o...

DOI :10.12659/MSM.935379

Med Sci Monit 2021; 27:e935379

13 Nov 2021 : Clinical Research  

Acceptance of COVID-19 Vaccination and Its Associated Factors Among Cancer Patients Attending the Oncology ...

DOI :10.12659/MSM.932788

Med Sci Monit 2021; 27:e932788

08 Mar 2022 : Review article  

A Review of the Potential Roles of Antioxidant and Anti-Inflammatory Pharmacological Approaches for the Man...

DOI :10.12659/MSM.936292

Med Sci Monit 2022; 28:e936292

01 Nov 2020 : Review article  

Long-Term Respiratory and Neurological Sequelae of COVID-19

DOI :10.12659/MSM.928996

Med Sci Monit 2020; 26:e928996

Your Privacy

We use cookies to ensure the functionality of our website, to personalize content and advertising, to provide social media features, and to analyze our traffic. If you allow us to do so, we also inform our social media, advertising and analysis partners about your use of our website, You can decise for yourself which categories you you want to deny or allow. Please note that based on your settings not all functionalities of the site are available. View our privacy policy.

Medical Science Monitor eISSN: 1643-3750
Medical Science Monitor eISSN: 1643-3750