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"Drugs that edit mRNA sequences are beginning to enter clinical trials to treat various genetic diseases. Many of these use a family of enzymes called ADARs, which can repair or disable defective genes by swapping individual bases in their mRNA transcripts. Like its high-profile relative CRISPR, mRNA editing uses engineered nucleotide strings to lure the enzymes to their target genetic sequences. At least ten companies have launched or are about to launch early stage trials to treat the hereditary disorder alpha-1 antitrypsin deficiency, which is caused by a single mutation that is easily reversible through mRNA editing. In preclinical studies, one company’s editing system fixed up to 75% or more of the defective proteins."
"Oligonucleotide-based drugs already come in many flavours. The newest of these now aims to edit mRNA one base at a time, by harnessing endogenous enzymes called adenosine deaminases acting on RNA (ADAR). ...
A key difference between DNA- and RNA-editing is the dosing schedule. Whereas CRISPR-based drugs aim to provide a one-time treatment, the ADAR pipeline is mostly comprised of redosable drugs that are given every few weeks or less frequently. Their effects, as a result, are reversible. ..."
RNA-editing drugs advance into clinical trials ADAR-based editors that can change the mRNA code offer new opportunities in both rare genetic diseases and common complex ones.
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