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A28 SCIENCE
Tuesday 10 March 2020
Doctors try 1st CRISPR editing in the body for blindness

By MARILYNN MARCHIONE
Scientists say they have
used the gene editing tool
CRISPR inside someone’s
body for the first time, a
new frontier for efforts
to operate on DNA, the
chemical code of life, to
treat diseases.
A patient recently had it
done at the Casey Eye In-
stitute at Oregon Health &
Science University in Port-
land for an inherited form of
blindness, the companies
that make the treatment
announced Wednesday.
They would not give details
on the patient or when the
surgery occurred.
It may take up to a month
to see if it worked to restore
vision. If the first few at-
tempts seem safe, doctors
plan to test it on 18 children
and adults.
“We literally have the po-
tential to take people
who are essentially blind
and make them see,” said
Charles Albright, chief sci- In this undated photo provided by the Oregon Health & Science University on Wednesday, March 4, 2020, Dr. Andreas Lauer, right,
entific officer at Editas prepares to perform the first-ever in vivo CRISPR gene edit procedure for the BRILLIANCE clinical trial at the school's Casey Eye
Medicine, the Cambridge, Institute in Portland, on a patient who had an inherited form of blindness.
M a s s a c h u s e t t s - b a s e d Associated Press
company developing the search is very high. work as it should. that and to ensure that the in the eye and does not
treatment with Dublin- The people in this study It’s done in an hour-long treatment cuts only where travel to other parts of the
based Allergan. “We think have Leber congenital surgery under general an- it’s intended to, Pierce said. body, so “if something
it could open up a whole amaurosis, caused by a esthesia. He has consulted for Editas goes wrong, the chance of
new set of medicines to go gene mutation that keeps Through a tube the width and helped test a gene harm is very small,” he said.
in and change your DNA.” the body from making a of a hair, doctors drip three therapy, Luxturna, that’s “It makes for a good first
Dr. Jason Comander, an protein needed to con- drops of fluid containing sold for a different type of step for doing gene editing
eye surgeon at Massachu- vert light into signals to the the gene editing machin- inherited blindness. in the body.”
setts Eye and Ear in Bos- brain, which enables sight. ery just beneath the retina, Some independent experts Although the new study
ton, another hospital that They’re often born with little the lining at the back of the were optimistic about the is the first to use CRISPR
plans to enroll patients in vision and can lose even eye that contains the light- new study. to edit a gene inside the
the study, said it marks “a that within a few years. sensing cells. “The gene editing ap- body, another company,
new era in medicine” using Scientists can’t treat it with “Once the cell is edited, proach is really exciting. Sangamo Therapeutics,
a technology that “makes standard gene therapy -- it’s permanent and that We need technology that has been testing zinc finger
editing DNA much easier supplying a replacement cell will persist hopefully will be able to deal with gene editing to treat meta-
and much more effective.” gene -- because the one for the life of the patient,” problems like these large bolic diseases.
Doctors first tried in-the- needed is too big to fit in- because these cells don’t genes,” said Dr. Jean Ben- Other scientists are using
body gene editing in 2017 side the disabled viruses divide, said one study lead- nett, a University of Penn- CRISPR to edit cells outside
for a different inherited dis- that are used to ferry it into er not involved in this first sylvania researcher who the body to try to treat can-
ease using a tool called cells. case, Dr. Eric Pierce at Mas- helped test Luxturna at the cer, sickle cell and some
zinc fingers. So they’re aiming to edit, sachusetts Eye and Ear. Children’s Hospital of Phila- other diseases.
Many scientists believe or delete the mutation by Doctors think they need to delphia. All of these studies have
CRISPR is a much easier making two cuts on either fix one tenth to one third of In one day, she had three been done in the open,
tool for locating and cut- side of it. The hope is that the cells to restore vision. In calls from families seeking with government regula-
ting DNA at a specific spot, the ends of DNA will recon- animal tests, scientists were solutions to inherited blind- tors’ approval, unlike a Chi-
so interest in the new re- nect and allow the gene to able to correct half of the ness. nese scientist’s work that
cells with the treatment, Al- “It’s a terrible disease,” she brought international scorn
bright said. said. “Right now they have in 2018.
The eye surgery itself poses nothing.” He Jiankui used CRISPR to
little risk, doctors say. Infec- Dr. Kiran Musunuru, another edit embryos at the time
tions and bleeding are rel- gene editing expert at the of conception to try to
atively rare complications. University of Pennsylvania, make them resistant to in-
One of the biggest poten- said the treatment seems fection with the AIDS virus.
tial risks from gene editing is likely to work, based on Changes to embryos’ DNA
that CRISPR could make un- tests in human tissue, mice can pass to future genera-
intended changes in other and monkeys. tions, unlike the work being
genes, but the companies Full Coverage: Health done now in adults to treat
have done a lot to minimize The gene editing tool stays diseases.q