genomemag | Imagine if doctors could correct a cataract, for
instance, not by using a scalpel or laser to perform surgery, but rather
by sending off miniature surgical tools to reach right in and fix the
diseased gene that was responsible. It might surprise you to learn that
scientists have already shown that this sort of thing is doable today —
not in humans perhaps, but in much tinier and fuzzier mice in the lab.
The procedure doesn’t work perfectly every time (which partly explains
why no one has tried it in a person just yet), but when it does, the
animals grow healthy and disease-free.
Scientists in China successfully cured 24 mice of their eye
condition, which was produced by a single, mutant copy of one gene. That
demonstration, reported in the scientific literature two years ago, was
billed as the first to show that it’s possible to correct a genetic
disease using a genome editing tool, which scientists call CRISPR-Cas9.
Although in mice, the findings offered the first proof of principle that
scientists and doctors might one day have sufficient skill and
precision to edit single-gene disorders out of human genomes in much the
same way.
Jinsong Li, one of the leaders of the study from the Chinese Academy
of Sciences, said then that he believes it is “absolutely possible to
use CRISPR to cure genetic disease in the near future.” As further
evidence in support of Li’s conclusion, his paper came out alongside
another by researchers in the Netherlands. They had used CRISPR to
correct a gene that causes cystic fibrosis in adult stem cells derived
from patients with the single-gene disorder.
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