We’ve all heard a lot about genetic modification this year, thanks in large part to one Chinese scientist taking it upon himself to edited the genes of human babies. His work was universally decried by fellow scientists and he got himself into some serious trouble because of it.
But genetic modification is an incredible tool that is showing promise in besting some really awful conditions, and it might not be long before scientists can selectively “fix” inherited diseases before they become a problem. A new study published in Cell Research offers hope for making conditions like Huntington’s disease a thing of the past.
Salk Institute researchers have invented a new method for editing genes that gives scientists greater flexibility in correcting genetic mutations that cause serious issues, including the mutations that lead to Huntington’s disease and the rapid-aging condition progeria.
The new tool is called SATI, and it’s based on the popular CRISPR-Cas9 gene-editing technique. The scientists developing SATI specifically designed it to work with a wide range of cell types that are often difficult to work with using existing gene-editing tools.
Rather than totally replacing troublesome genes — and risking any number of potential side effects that could result — scientists using SATI insert a known good copy of a misbehaving gene into a specific region of the DNA. The gene is then incorporated into the genome thanks to the DNA’s natural repair functions, effectively correcting the mutation without the possible risks of whole gene replacement.
“We sought to create a versatile tool to target these non-coding regions of the DNA, which would not affect the function of the gene, and enable the targeting of a broad range of mutations and cell types,” Mako Yamamoto, co-first author of the research, said in a statement. “As a proof-of-concept, we focused on a mouse model of premature aging caused by a mutation that is difficult to repair using existing genome-editing tools.”
In their testing, the SATI development team used the new technique to correct the gene responsible for progeria in mice. The results were dramatic, with the rodents showing clear signs that the age-affecting syndrome had been curbed, increasing the lifespan of the animals by 45 percent. If something similar could be done for humans with progeria, that would translate to over a decade of additional life, the researchers say.
Going forward, the team plans to continue the development of the tool and increase its efficiency, boosting gains from the gene-editing procedure and potentially showing even more dramatic results.