Not exact matches
But, as journalist Steve Connor reports, the reference to editing was intentional: «Scientists have used the genome - editing technology to cure adult laboratory
mice of an inherited liver disease by
correcting a single «letter» of the genetic alphabet which had been mutated
in a vital
gene involved
in liver metabolism.»
The researchers
corrected a mutation
in liver cells
in mice by snipping out the
gene and sewing
in a healthy copy of it.
The UT Southwestern group had previously used CRISPR - Cas9, the original
gene - editing system, to
correct the Duchenne defect
in a
mouse model of the disease and
in human cells.
Using the new
gene - editing enzyme CRISPR - Cpf1, researchers at UT Southwestern Medical Center have successfully
corrected Duchenne muscular dystrophy
in human cells and
mice in the lab.
UT Southwestern Medical Center researchers successfully used a new
gene editing method to
correct a mutation that leads to Duchenne muscular dystrophy (DMD)
in a
mouse model of the condition.
In a study of mice, they found that they could correct the mutated gene that causes a rare liver disorder, in 6 percent of liver cells — enough to cure the mice of the disease, known as tyrosinemi
In a study of
mice, they found that they could
correct the mutated
gene that causes a rare liver disorder,
in 6 percent of liver cells — enough to cure the mice of the disease, known as tyrosinemi
in 6 percent of liver cells — enough to cure the
mice of the disease, known as tyrosinemia.
In an independent effort, they introduced progressively smaller pieces of DNA from the large region known to contain the gene into embryos of the mutant mice, looking for the smallest piece that would correct the mutation in adult mice and restore a normal rhyth
In an independent effort, they introduced progressively smaller pieces of DNA from the large region known to contain the
gene into embryos of the mutant
mice, looking for the smallest piece that would
correct the mutation
in adult mice and restore a normal rhyth
in adult
mice and restore a normal rhythm.
Results showed that naturally healthy macrophages and
gene -
corrected macrophages worked equally well
in correcting the disease
in the
mice.
The research, the cover story of this month's Science Advances, builds upon previous studies from Dr. Olson
in which CRISPR - Cas9
corrected a single
gene mutation that caused DMD
in mice.
To
correct RPE65 mutation damage, scientists are evaluating drugs, cell transplants and
gene delivery
in Rpe65 knockout, transgenic and spontaneous mutant
mice, and spontaneous Rpe65 - mutant dogs.
Bethesda, Md., Wed., Oct. 26, 2016 - For the first time, National Institutes of Health (NIH) researchers have demonstrated
in mice that
gene therapy may be the best method for
correcting the single faulty
gene that causes Niemann - Pick disease, type C1 (NPC1).
For the first time, National Institutes of Health (NIH) researchers have demonstrated
in mice that
gene therapy may be the best method for
correcting the single faulty
gene that causes Niemann - Pick disease, type C1 (NPC1).