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THE DAILY ASTORIAN • THuRSDAY, FEbRuARY 7, 2019
Tests suggest scientists achieved 1st ‘in body’ gene editing
By MARILYNN
MARCHIONE
Associated Press
Scientists think they have
achieved the first gene edit-
ing inside the body, altering
DNA in adults to try to treat
a disease, although it’s too
soon to know if this will help.
Preliminary results sug-
gest that two men with a rare
disorder now have a correc-
tive gene at very low levels,
which may not be enough to
make the therapy a success.
Still, it’s a scientific mile-
stone toward one day doctor-
ing DNA to treat many dis-
eases caused by faulty genes.
“This is a first step,” said
Dr. Joseph Muenzer of the
University of North Carolina
at Chapel Hill, who helped
test the treatment. “It’s just
not potent enough.”
He gave the results today
at a conference in Orlando,
Florida, and has consulted
for the therapy’s maker, Cal-
ifornia-based Sangamo Ther-
apeutics. Researchers are
working on a stronger ver-
sion of the treatment.
Gene editing is intended
as a more precise way to do
gene therapy, to disable a bad
gene or supply a good one
that’s missing. Trying it in
AP Photo/Eric Risberg
Brian Madeux starts to receive the gene editing therapy for Hunter syndrome in 2017.
adults to treat diseases is not
controversial and the DNA
changes do not pass to future
generations, unlike the recent
case of a Chinese scientist
who claims to have edited
twin girls’ genes when they
were embryos.
Sangamo’s
studies
involve men with Hunter
or Hurler syndrome, dis-
eases caused by a missing
gene that makes an enzyme
to break down certain sugar
compounds. Without it, sug-
ars build up and damage
organs, often killing people
in their teens.
In 2017, Brian Madeux of
Arizona became the first per-
son to try it. Through an IV,
he received many copies of a
corrective gene and an edit-
ing tool called zinc finger
nucleases to insert it into his
DNA.
Results on him and seven
other Hunter patients, plus
three with Hurler syndrome,
suggest the treatment is safe,
which was the main goal
of these early experiments.
Three problems — bronchi-
tis, an irregular heartbeat and
a hernia — were deemed
due to the diseases, not the
treatment.
Tissue samples showed
evidence of gene editing at
very low levels in two Hunter
patients who were given a
middle dose but not in one
given a low dose. Tests are
expected later this year on
patients who received the
highest dose and on Hurler
patients.
Blood tests detected
slightly higher levels of the
missing enzyme in a few of
the Hunter patients but none
of them reached normal lev-
els. One patient had a larger
increase but also showed
signs that his immune sys-
tem might be attacking the
therapy. He was treated for
that and symptoms resolved.
More encouraging results
were seen in Hurler patients
— enzyme levels rose to nor-
mal in all three after treat-
ment, tests on certain blood
cells showed.
“This is very promis-
ing” for Hurler patients, said
Dr. Paul Harmatz of UCSF
Benioff Children’s Hospi-
tal Oakland, who presented
those results.
None of the patients with
either disease showed a sus-
tained decline in urine lev-
els of the troubling sugar
compounds, though, and
some other tests also did not
detect intended effects of the
therapy.
The key test will be stop-
ping the patients’ weekly
enzyme treatments to see if
their bodies can now make
enough of it on their own.
Three have gone off treat-
ments so far and one was
recently advised to resume
them because of fatigue and
rising levels of the sugar
compounds. The others have
not been off long enough to
know how they will fare.
“It looks like it’s safe ...
that’s a very positive sign,”
said one independent expert,
Dr. Kiran Musunuru of the
University of Pennsylvania.
He called the early results
promising but said “it’s hard
to be sure it’s doing any
good” until patients are stud-
ied longer.
“What they’re trying to
do with gene editing is very
challenging,” he said. “It’s
much harder to make a cor-
rection or insert a gene” than
to disable one.
Dr. Tyler Reimschisel
of Vanderbilt University
agreed.
“It’s not discouraging,
it’s just early and on a small
amount of people,” he said.
“This is definitely a novel
and innovative treatment”
but it’s not clear if it’s going
to help.
WORLD IN BRIEF
Wednesday show the rocket was gone
with what appears to be burn marks on its
launch pad.
It wasn’t immediately clear if the satel-
lite, if launched, made it into orbit.
The U.S. alleges such launches defy a
U.N. Security Council resolution calling
on Iran to undertake no activity related
to ballistic missiles capable of delivering
nuclear weapons.
Associated Press
Democrats seek Green New
Deal for climate change
WASHINGTON — Democrats includ-
ing Rep. Alexandria Ocasio-Cortez of
New York are calling for a Green New
Deal intended to transform the U.S. econ-
omy to combat climate change and create
thousands of jobs in renewable energy.
The freshman lawmaker and vet-
eran Sen. Ed Markey of Massachusetts
are teaming up on the plan, which aims
to eliminate the U.S. carbon footprint by
2030.
While setting lofty goals, the plan does
not explicitly call for eliminating the use
of fossil fuels such as oil and natural
gas, a nod to pragmatism that may disap-
point some of Ocasio-Cortez’s strongest
supporters.
Even so, their Green New Deal goes far
beyond the Clean Power Plan proposed by
President Barack Obama. President Don-
ald Trump has scrapped Obama’s plan,
which imposed emissions limits on coal-
fired power plants, as a job-killer.
Images suggest Iran
launched satellite
DUBAI, United Arab Emirates — Iran
appears to have attempted a second satel-
lite launch despite U.S. criticism that its
space program helps the country develop
ballistic missiles, satellite images released
today suggest. Iran did not immediately
acknowledge conducting such a launch.
The images show a rocket at the Imam
Khomeini Space Center in Iran’s Sem-
nan province on Tuesday. Images from
Dislike: Facebook will appeal
German data-use ruling
AP Photo/Jeff Chiu
San Francisco firefighters battle a fire after a gas explosion.
San Francisco gas explosion
shoots fire that burns buildings
SAN FRANCISCO — A gas explo-
sion in a San Francisco neighbor-
hood shot flames into the air for hours
Wednesday and burned five buildings,
sending panicked residents and workers
fleeing into the streets.
It took utility crews more than two
hours to put out the fire after private con-
struction workers cut a natural gas line,
igniting the towering flames. Authorities
initially said five workers were missing,
but the entire construction crew was found
safe, and no other injuries were reported.
SCHEDULE
THE DAILY
ASTORIAN
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6 PM
Officials evacuated several nearby
buildings, including a medical clinic and
apartment buildings. Vehicles on a busy
street got rerouted as authorities cor-
doned off the bustling retail and residen-
tial neighborhood.
Pacific Gas & Electric is under
heightened scrutiny over its natural gas
pipelines after one exploded under a
neighborhood south of San Francisco
in 2010, killing eight people and wip-
ing out a neighborhood in suburban San
Bruno.
BERLIN — German authorities ruled
today that Facebook should not be allowed
to use customer data from other apps and
websites to help target advertisements
shown on their Facebook pages with-
out their explicit consent, saying it was
exploiting its dominant position in social
media.
The Federal Cartel Office, or
Bundeskartellamt, said Facebook was
guilty of “exploitative abuse” by forcing
users to agree to allow it to collect data
from other Facebook-owned services
like WhatsApp and Instagram, as well as
third-party websites through the “Like” or
“Share” features.
Facebook said it rejected the decision,
and would appeal within the one-month
frame before it becomes final.
If the ruling is upheld, Facebook will
be required to allow users to specifically
approve data collected from other Face-
book-owned sources and third-party web-
sites be assigned to their accounts. The
decision is not about Facebook’s pro-
cessing of data generated by its own site,
which the Cartel Office acknowledged is
the business model for data-based social
networks like Facebook.
Evening listings
THURSDAY
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