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These modifications regulate geneexpression without changing the sequence or structure of DNA. The tool could also prove to be safer than conventional CRISPR-based gene therapies as it does not involve DNA editing, and thus would not cause potentially harmful off-target genomic changes. pyogenes dCas9.
InteRNA is focusing on the development of gene-silencing drugs based on microRNA, naturally occurring, non-coding strands of RNA that are thought to regulate geneexpression in cells.
These modifications regulate geneexpression without altering the sequence or structure of DNA. The tool could also prove to be safer than conventional CRISPR-based gene therapies as it does not involve DNA editing, and thus would not cause potentially harmful off-target genomic changes. pyogenes dCas9.
Coding RNAs include messenger RNA (mRNA) and short interfering RNA (siRNA), which encode proteins and silencegeneexpression, respectively. RNA interference (RNAi) Therapeutics : Fundamentally, RNAi is a natural process of post-transcriptional genesilencing, involving short strands of nucleic acids.
For example, “genesilencing” is a less-than-ideal description for the mechanism of RNA interference (RNAi) therapeutics. Although this approach “silences” the output of genes (by disrupting messenger RNA [mRNA]), the lack of specificity suggests the possibility of direct action on genes in the nucleus.
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