MOA animation exploring RNA editing and RNA's role in genetic diseases.
This mechanism-of-action animation is created for a global biotechnology company, whose mission is to engineer precision genetic medicine for rare diseases. The animation explores RNA and its role in genetic disease and an RNA editing platform that involves gene editing when processing the pre-mRNA.
Gene Editing (also called genome editing) is a group of technologies that give scientists the ability to change an organism's DNA. These technologies allow genetic material to be added, removed, or altered at particular locations in the genome. Several approaches to genome editing have been developed, such as CRISPR-Cas9 (clustered regularly interspaced short palindromic repeats and CRISPR-associated protein 9).
RNA editing (RNA modification) is the posttranscriptional modification of an RNA nucleotide sequence at one or more positions. There are two general types of RNA editing - substitution editing, and insertion/deletion editing. RNA editing of either type leads to the formation of transcripts whose sequence differs from that of the genome template. Such RNA sequence differences between mature transcript and encoding genome represent a form of genetic recoding.
RNA editing is widely observed in eukaryotic organisms and their viruses. Editing, like splicing, represents a form of processing that has the capacity to amplify genetic diversity and alter gene product function by modifying the information transfer process at the posttranscriptional level.
Unlike DNA editing, which is permanent, the effects of RNA editing are reversible, since cells are constantly churning out new copies of RNA.
Reference: (1) medlineplus.gov, (2) RNA Editing, Charles E. Samuel, in Reference Module in Biomedical Sciences, 2019, (3) cen.acs.org
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