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Scientists Break Ground In Use of Gene-Editing Tool

CRISPR was employed against a patient's HIV
By Newser Editors and Wire Services
Posted Sep 11, 2019 6:55 PM CDT
A 2011 electron microscope image made available by the Centers for Disease Control and Prevention shows HIV virions.   (Maureen Metcalfe, Tom Hodge/CDC via AP)

(Newser) – Scientists are reporting the first use of the gene-editing tool CRISPR to try to cure a patient's HIV infection by providing blood cells that were altered to resist the AIDS virus. The gene-editing tool has long been used in research labs, and a Chinese scientist was scorned last year when he revealed he used it on embryos that led to the birth of twin girls. Editing embryos is considered too risky, partly because the DNA changes can pass to future generations. Wednesday's report in the New England Journal of Medicine, by different Chinese researchers, is the first published account of using CRISPR to treat a disease in an adult, where the DNA changes are confined to that person. The attempt was successful in some ways but fell short of being an HIV cure, the AP reports.

Still, it shows that gene editing holds promise and seems precise and safe in this patient so far, said Dr. Carl June, a University of Pennsylvania genetics expert who wrote a commentary in the journal. "That's really good for the field," June said. The research was done openly with advance notice on a scientific registry and standard informed consent procedures. Some of those steps were missing or questioned in last year's embryo work. Gene editing permanently alters DNA. CRISPR is a relatively new tool scientists can use to cut DNA at a specific spot. One encouraging result: Multiple tests show that the editing did not have unintended effects on other genes. "They did a very innovative experiment on a patient, and it was safe," said an expert at Johns Hopkins, per Live Science.

(Read more CRISPR stories.)

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