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Abstract

October - December 2023 | Vol. 16 | Issue 4 | 08-13
RNA-based Therapies for Monogenic Disorders
Sheeba Farooqui, Shreya S Shetty
Department of Medical Genetics, Kasturba Medical College, Manipal Academy of Higher Education, Manipal, Karnataka, India
Address for Correspondence Email: sheebafarooqui@gmail.com
 
Abstract
Monogenic disorders are conditions caused by variants in single genes. The current treatment strategies for monogenic disorders include DNA, RNA, or protein-based therapies. RNA-based therapeutics can be divided into four main categories namely antisense oligonucleotides, RNA interference, aptamers, and messenger RNA (mRNA) based therapies. Here, we discuss the RNA therapies that have emerged as promising strategies for the treatment of several monogenic disorders in recent years.
 
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