Despite the hype and future for gene editing, it’s a risky business full of ethical dilemmas. Researchers from UC Berkeley and the University of California, San Diego took the ethics to heart and focused on using CRIPSR-Cas9 to alter the product of those genes.
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CRISPR might need a crash course to explain: DNA is double stranded and makes single-stranded RNA. RNA in turn, makes proteins. If you edit the DNA, the proteins are also changed, permanently. If you edit the RNA, however, the basic workings of DNA remain the same, while the proteins can be safely manipulated. That means the level of gene expression can be turned up or turned down. Theoretically, RNA editing is also reversible. It also doesn’t change the genome, meaning changes don’t get passed down.
Gene Yeo is a professor of cellular and molecular at USCD and the team lead showed that this team could target RNA in living cells, which is the very first step in being able to treat diseases like neurodegeneration and muscular dystrophy.
“We had to show that [RNA targeting] was feasible in living cells…“Now, we are trying many different ways of improving it.”
The research was presented in March’s edition of Cell.
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