A new version of a gene-editing system is the smallest yet — which may make it ideal for tweaking the genome of living organisms.
The system, derived from the defenses of single-celled organisms called archaea, is similar to the famous CRISPR-Cas9 gene editing tool.
CRISPR-Cas9 is derived from bacteria and is made up of two parts: a guide RNA that can bind to DNA, and Cas9, an enzyme that can slice the DNA at the spot the guide RNA targets. Bacteria and archaea use these systems to destroy invading viruses, but with the right bioengineering techniques, these tools can be used to turn genes on and off in humans and even alter genes by changing their sequences.
The ultimate goal is to cure genetic diseases by snipping out dysfunctional genes and replacing them with sequences that work properly. That idea is already being tested in humans: Clinical trials using CRISPR-Cas9 to cure a condition called Leber’s congenital amaurosis 10 (LCA10), which causes blindness, started in March 2020...
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