The ability to alter our genome through CRISPR has a multitude of implications for treating genetic diseases. However, experimental gene editing with some kinds of CRISPR machinery has proven to have unintended consequences, such as unexpected deletions and hostile host immune response. Additionally, public perception to altering an individual's genome has added a layer of contention to the use of CRISPR. In the past few years, more scientists have started to look at the potential of RNA editing as an alternative.
RNA editing works by utilizing enzymes called ADARs (adenosine deaminases) which naturally occur in human cells and are capable of acting on double stranded RNA to change adenosine nucleotides to inosine (a nucleoside which often occurs in tRNAs and is read as guanosine during translation). The functional purpose of this change is still relatively unknown, as the editing usually occurs on non-coding strands, but research in mice have shown ADAR mutations can result in non-viable offspring, suggesting an important role for this class of proteins.
ADARs and other RNA editing proteins could potentially be used for clinical applications such as altering mRNA codons and allowing for production of a missing protein. Experimental studies on mice with muscular dystrophy showed recovery of about 5% normal levels of dystrophin protein which had previously been completely missing. However, researchers are still uncovering the drawbacks and benefits of RNA editing. While it wouldn't carry the same risks as CRISPR as the enzymes do not work directly on the genome, RNA editing is likely to perform less efficiently and be limited in function in ways that CRISPR is not. Though much is still unknown, RNA editing is a field with a lot of potential for future applications to genetic and clinical research.
Audrey Tjahjadi
Link to original article: https://www.nature.com/articles/d41586-020-00272-5
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