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



04 June 2022 : Editorial  

Editorial: Acute Hepatitis of Unknown Origin in Children. Is Autoimmunity at Play?

Nahum Mendez-Sanchez, Shreya C. Pal
Faculty of Medicine, National Autonomous University of Mexico, Mexico City, Mexico

DOI: 10.12659/MSM.937371

Med Sci Monit 2022; 28:e937371


27 June 2022 : Clinical Research  

Shoulder Injury Related to Vaccine Administration (SIRVA) in 16 Patients Following COVID-19 Vaccination Who...

Med Sci Monit In Press; DOI: 10.12659/MSM.937430  

27 May 2022 : Clinical Research  

Factors Associated with Falls During Hospitalization for Coronavirus Disease 2019 (COVID-19)

Med Sci Monit 2022; 28:e936547

In Press

28 Jun 2022 : Meta-Analysis  

Urodynamics in Early Diagnosis of Diabetic Bladder Dysfunction in Women: A Systematic Review and Meta-Analysis

Med Sci Monit In Press; DOI: 10.12659/MSM.937166  

28 Jun 2022 : Clinical Research  

Serum Calprotectin – a NET Product – as a Biomarker of Disease Activity in Patients with Systemic Lupus Ery...

Med Sci Monit In Press; DOI: 10.12659/MSM.936534  

28 Jun 2022 : Database Analysis  

LINC00963 May Be Associated with a Poor Prognosis in Patients with Cervical Cancer

Med Sci Monit In Press; DOI: 10.12659/MSM.935070  

27 Jun 2022 : Clinical Research  

Shoulder Injury Related to Vaccine Administration (SIRVA) in 16 Patients Following COVID-19 Vaccination Who...

Med Sci Monit In Press; DOI: 10.12659/MSM.937430  

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

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

01 Jan 2022 : Editorial  

Editorial: Current Status of Oral Antiviral Drug Treatments for SARS-CoV-2 Infection in Non-Hospitalized Pa...

DOI :10.12659/MSM.935952

Med Sci Monit 2022; 28:e935952

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