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RNA Editing - Wave Life Sciences
November 13 , 2024
7 hrs 0 min
40
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- Biotechnology company Wave Life Sciences is becoming the first company to treat a genetic condition by editing RNA at the clinical level.
- The role of RNA in a function called RNA interference has been essential for the success of CRISPR-Cas9 gene-editing.
- The Cells synthesise messenger RNA (mRNA) using instructions in DNA and then ‘read’ instructions from the mRNA to make functional proteins.
- RNA editing allows the scientists to fix mistakes in the mRNA after the cell has synthesised it but before the cell reads it to make the proteins.
- Wave Life Sciences used RNA editing to treat α-1 antitrypsin deficiency (AATD), an inherited disorder.
- RNA editing has some advantages over DNA editing, especially on safety and the flexibility.
- DNA editing makes permanent changes to a person’s genome and sometimes this can lead to irreversible errors.
- But RNA editing makes temporary changes, allowing the effects of the edits to fade over time.
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