Oct 11, 2019

CRISPR used to treat first U.S. patient with genetic disorder

Photo: Gregor Fischer/picture alliance/Getty Images

Doctors are using CRISPR to treat a woman with sickle cell disease, the first U.S. patient with a genetic disorder to receive such treatment, NPR reports.

The big picture: Advances in gene editing technology have given new hope to people with diseases that not long ago seemed untreatable.

  • But these new therapies are still experimental, and the technologies also carry the potential to be dangerously misused.

Details: The woman received infusions of genetically modified bone marrow cells, which will hopefully give her healthy red blood cells.

  • The companies sponsoring the study said earlier this year that they'd used CRISPR to treat a similar blood disorder in Germany, and there's been some evidence that the treatment may be working.
  • Doctors are also experimenting with treating cancer patients with CRISPR.

Go deeper: Genetic technology's double-edged sword

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Doctors safely use CRISPR on cancer patients

A researcher performs a CRISPR/Cas9 process at the Max-Delbrueck-Centre for Molecular Medicine. Photo: Gregor Fischer/picture alliance via Getty Images

Three cancer patients in the U.S. were the first to be safely injected with the gene editing tool CRISPR as a form of immunotherapy to fight their illness, AP reports.

Reality check: It's too soon to know whether the treatment will help, doctors say. The patients, who all failed multiple standard treatments, had varying results after two to three months.

Go deeperArrowNov 7, 2019

New advance could broaden use of CRISPR gene editing

Illustration: Sarah Grillo/Axios

Scientists announced Monday they have developed a "versatile and precise new approach" to targeting and replacing mutated genes that could eventually be used to address roughly 89% of genetic variations known to play a role in human diseases.

Why it matters: The technique, called prime editing, adds a third editing tool for mammalian cells in a manner that lessens the rate of the dangerous off-target deletions or insertions caused by the other types of tools, according to Broad Institute's David Liu, co-author of the paper published in Nature.

Go deeperArrowOct 22, 2019

Genetic testing may prevent ER visits for kids with Type I diabetes

Researchers at the University of Virginia are developing genetic testing to keep children with undiagnosed Type I diabetes out of emergency rooms, NPR reports.

Why it matters: Almost 50% of all children who develop Type 1 diabetes end up hospitalized in a coma because they didn't know they had the autoimmune disease, per the report.

Go deeperArrowOct 15, 2019