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RNA Therapeutics: A Novel Approach to Treating Diseases

Roots Analysis

Coding RNAs include messenger RNA (mRNA) and short interfering RNA (siRNA), which encode proteins and silence gene expression, respectively. ASOs are short single-stranded nucleotides that bind to specific messenger RNAs and prevent the production of a particular protein.

RNA 40
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The use of base editing in stem-cell based therapies

Drug Discovery World

A second generation CRISPR-Cas gene editing system, base editing, reduces the risk of potentially deleterious effects associated with other gene editing technologies, presenting a safe and effective means of editing cells for therapeutic applications. Dis Model Mech. 2020;13:dmm042317. doi:10.1242/dmm.042317. Nat Commun. 2020;11:810.