East Oregonian : E.O. (Pendleton, OR) 1888-current, August 03, 2017, Page Page 7A, Image 7

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    NATION/WORLD
Thursday, August 3, 2017
East Oregonian
Page 7A
Embryo gene-repair holds promise for inherited disease
OHSU researchers
fi rst to repair gene that
causes heart defects
WASHINGTON
(AP)
—
Altering human heredity? In a
fi rst, researchers safely repaired
a disease-causing gene in human
embryos, targeting a heart defect
best known for killing young
athletes — a big step toward one
day preventing a list of inherited
diseases.
In a surprising discovery, a
research team led by Oregon
Health and & Science University
reported Wednesday that embryos
can help fi x themselves if scien-
tists jump-start the process early
enough.
It’s laboratory research only,
nowhere near ready to be tried in
a pregnancy. But it suggests that
scientists might alter DNA in a way
that protects not just one baby from
a disease that runs in the family,
but his or her offspring as well.
And that raises ethical questions.
“I for one believe, and this paper
supports the view, that ultimately
gene editing of human embryos
can be made safe. Then the ques-
tion truly becomes, if we can do it,
should we do it?” said Dr. George
Daley, a stem cell scientist and
dean of Harvard Medical School.
He wasn’t involved in the new
research and praised it as “quite
remarkable.”
“This is defi nitely a leap
forward,” agreed developmental
geneticist Robin Lovell-Badge of
Britain’s Francis Crick Institute.
Today, couples seeking to avoid
passing on a bad gene sometimes
have embryos created in fertility
clinics so they can discard those
that inherit the disease and attempt
pregnancy only with healthy ones,
if there are any.
Gene editing in theory could
rescue diseased embryos. But
so-called “germline” changes —
altering sperm, eggs or embryos
— are controversial because they
would be permanent, passed down
to future generations. Critics worry
about attempts at “designer babies”
instead of just preventing disease,
and a few previous attempts at
learning to edit embryos, in China,
didn’t work well and, more impor-
tantly, raised safety concerns.
In a series of laboratory exper-
iments reported in the journal
Kristyna Wentz-Graff/Oregon Health & Science University via AP
In this July 31 photo provided by Oregon Health & Science University, Shoukhrat Mitalipov, left,
talks with research assistant Hayley Darby in the Mitalipov Lab at OHSU in Portland. Mitalipov led
a research team that, for the fi rst time, used gene editing to repair a disease-causing mutation in
human embryos, laboratory experiments that might one day help prevent inherited diseases from
being passed to future generations.
Oregon Health & Science University via AP
In this microscope photo provided by Oregon Health & Science
University, human embryos grow in a laboratory for a few days
after researchers repair a heart disease-causing genetic mutation.
Nature, the Oregon researchers
tried a different approach.
They targeted a gene mutation
that causes a heart-weakening
disease, hypertrophic cardiomy-
opathy, that affects about 1 in 500
people. Inheriting just one copy of
the bad gene can cause it.
The team programmed a
gene-editing tool, named CRIS-
PR-Cas9, that acts like a pair of
molecular scissors to fi nd that
mutation — a missing piece of
genetic material.
Then came the test. Researchers
injected sperm from a patient
with the heart condition along
with those molecular scissors into
healthy donated eggs at the same
time. The scissors cut the defective
DNA in the sperm.
Normally cells will repair a
CRISPR-induced cut in DNA by
essentially gluing the ends back
together. Or scientists can try
delivering the missing DNA in a
repair package, like a computer’s
cut-and-paste program.
Instead, the newly forming
embryos made their own perfect fi x
without that outside help, reported
Oregon Health & Science Univer-
sity senior researcher Shoukhrat
Mitalipov.
We all inherit two copies of
each gene, one from dad and one
from mom — and those embryos
just copied the healthy one from
the donated egg.
“The embryos are really looking
for the blueprint,” Mitalipov, who
directs OHSU’s Center for Embry-
onic Cell and Gene Therapy, said
in an interview. “We’re fi nding
embryos will repair themselves if
you have another healthy copy.”
It worked 72 percent of the time,
in 42 out of 58 embryos. Normally
a sick parent has a 50-50 chance of
passing on the mutation.
Previous
embryo-editing
attempts in China found not every
cell was repaired, a safety concern
called mosaicism. Beginning
the process before fertilization
avoided that problem: Until now,
“everybody was injecting too late,”
Mitalipov said.
Nor did intense testing uncover
any “off-target” errors, cuts to
DNA in the wrong places, reported
the team, which also included
researchers from the Salk Institute
for Biological Studies in Cali-
fornia and South Korea’s Institute
for Basic Science. The embryos
weren’t allowed to develop beyond
eight cells, a standard for labora-
tory research.
The experiments were privately
funded; U.S. tax dollars aren’t
allowed for embryo research.
Genetics and ethics experts
not involved in the work say it’s
a critical fi rst step — but just one
step — toward eventually testing
the process in pregnancy, some-
thing currently prohibited by U.S.
policy.
“This is very elegant lab work,”
but it’s moving so fast that society
needs to catch up and debate
how far it should go, said Johns
Hopkins University bioethicist
Jeffrey Kahn.
And lots more research is
needed to tell if it’s really safe,
added Britain’s Lovell-Badge. He
and Kahn were part of a National
Academy of Sciences report
earlier this year that said if germ-
line editing ever were allowed, it
should be only for serious diseases
with no good alternatives and done
with strict oversight.
“What we do not want is for
rogue clinicians to start offering
treatments” that are unproven,
as has happened with some other
experimental technologies, he
stressed.
Among key questions: Would
the technique work if mom, not
dad, harbored the mutation? Is
repair even possible if both parents
pass on a bad gene?
Mitalipov is “pushing a fron-
tier,” but it’s responsible basic
research that’s critical for under-
standing embryos and disease
inheritance, noted University of
Pittsburgh professor Kyle Orwig.
In fact, Mitalipov said the
research should offer critics some
reassurance: If embryos prefer
self-repair, it would be extremely
hard to add traits for “designer
babies” rather than just eliminate
disease.
“All we did is un-modify the
already mutated gene.”
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Unwanted record: Biggest ever
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dead zone in Gulf of Mexico
NEW ORLEANS (AP) —
There’s an unwanted record
in the Gulf of Mexico: This
year’s “dead zone ,” a largely
human-caused phenomenon
where there’s too little oxygen
to support marine life, is the
biggest ever measured.
The low-oxygen, or
hypoxic , zone covers 8,776
square miles — about the
size of New Jersey, the
National Oceanic and Atmo-
spheric Administration said
Wednesday. The area is more
than 3 percent larger than the
2002 dead zone.
“We predicted it would
be large, and it is large,” said
scientist Nancy Rabalais, who
has been measuring the dead
zone since 1985.
She said the area was
actually larger, but the July
mapping cruise had to stop
before reaching the western
edge.
“The structure of the water
column was changing, so I’m
not sure how much larger
it would have been,” said
Rabalais, of the Louisiana
Universities Marine Consor-
tium .
Rabalais said winds from
the west and southwest appar-
ently had also compressed the
eastern half of the low-oxygen
area closer to shore than she’d
ever seen it. Without those
winds, it probably would
have covered a broader area,
she said.
Studies in the spring had
predicted the third-largest
dead zone ever — nearly
8,200 square miles.
Those studies are based
on examining nitrogen and
phosphorus in the Mississippi
River. The nutrients, which get
carried down from the river,
feed plankton blooms that die
and sink to the bottom, where
their decay uses oxygen.
The smallest measure-
ments were during or after
droughts: 1,696 square miles
in 2000 and 15 square miles
in 1988.
“This large dead zone size
shows that nutrient pollution,
primarily from agriculture and
developed land runoff in the
Mississippi River watershed
is continuing to affect the
nation’s coastal resources and
habitats in the Gulf,” NOAA
said in a news release.
The solar eclipse is coming
August 21
Come get the coolest eclipse
glasses in town at your nearest
East Oregonian office, or come
visit us at our Umatilla County
Fair booth, Aug. 8-12.
D
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$1 EACH
Best deal in town!
Pick some up for your friends
and family while supplies last.
East Oregonian
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