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Title: Scientists Edit Gene in 15 Patients That May Permanently Reduce High
Cholesterol

Link: https://science.slashdot.org/story/25/11/08/2...

A CRISPR-based drug given to study participants by infusion is raising hopes
for a much easier way to lower cholesterol, reports CNN: With a snip of a
gene, doctors may one day permanently lower dangerously high cholesterol,
possibly removing the need for medication, according to a new pilot study
published Saturday in the New England Journal of Medicine. The study was
extremely small - only 15 patients with severe disease - and was meant to
test the safety of a new medication delivered by CRISPR-Cas9, a biological
sort of scissor which cuts a targeted gene to modify or turn it on or off.
Preliminary results, however, showed nearly a 50% reduction in low-density
lipoprotein, or LDL, the "bad" cholesterol which plays a major role in heart
disease - the No.1 killer of adults in the United States and worldwide. The
study, which will be presented Saturday at the American Heart Association
Scientific Sessions in New Orleans, also found an average 55% reduction in
triglycerides, a different type of fat in the blood that is also linked to an
increased risk of cardiovascular disease. "We hope this is a permanent
solution, where younger people with severe disease can undergo a 'one and
done' gene therapy and have reduced LDL and triglycerides for the rest of
their lives," said senior study author Dr. Steven Nissen, chief academic
officer of the Sydell and Arnold Miller Family Heart, Vascular & Thoracic
Institute at Cleveland Clinic in Ohio.... Today, cardiologists want people
with existing heart disease or those born with a predisposition for hard-to-
control cholesterol to lower their LDL well below 100, which is the average
in the US, said Dr. Pradeep Natarajan, director of preventive cardiology at
Massachusetts General Hospital and associate professor of medicine at Harvard
Medical School in Boston... People with a nonfunctioning ANGPTL3 gene - which
Natarajan says applies to about 1 in 250 people in the US - have lifelong
levels of low LDL cholesterol and triglycerides without any apparent negative
consequences. They also have exceedingly low or no risk for cardiovascular
disease. "It's a naturally occurring mutation that's protective against
cardiovascular disease," said Nissen, who holds the Lewis and Patricia Dickey
Chair in Cardiovascular Medicine at Cleveland Clinic. "And now that CRISPR is
here, we have the ability to change other people's genes so they too can have
this protection." "Phase 2 clinical trials will begin soon, quickly followed
by Phase 3 trials, which are designed to show the effect of the drug on a
larger population, Nissen said." And CNN quotes Nissen as saying "We hope to
do all this by the end of next year. We're moving very fast because this is a
huge unmet medical need - millions of people have these disorders and many of
them are not on treatment or have stopped treatment for whatever reason."

Read more of this story at Slashdot.

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