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Updated: May 13, 2026

A Simple Alternative to Stereotactic Injection for Brain Specific Knockdown of miRNA
Published on: December 26, 2015
miRNA-Based Therapeutic Strategies
Masaharu Ishida1, Florin M Selaru
1Division of Gastroenterology and Hepatology School of Medicine Johns Hopkins University 720 Rutland Avenue, Ross 950, Baltimore, MD 21205 (410) 614-3811.
Abstract:
Micro-RNAs (miRNAs) are short non-coding RNA species, thought to act primarily through downregulation of target mRNA species with subsequent decrease in encoded proteins. Recent studies revealed that miRNAs play pivotal roles in physiology and disease, and therapeutic targeting has started being investigated. Generally, the up-regulation of miRNAs is achieved through administration of synthetic miRNAs or administration of miRNA expressing vectors. The down-regulation of miRNAs is achieved through administration of anti-sense nucleotides, often chemically modified to ensure stability and specificity. There are multiple potential limitations associated with the development and testing of miRNAs-based therapeutics. These issues include, but are not limited to, off-target effect, avoidance from internal nucleases, and toxicity for miRNA therapy. In this review, we will discuss recent advances in miRNA based therapeutic strategies.
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