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CRISPR Excitement

Researchers and drug companies are enthusiastic about the use of genome editing tools like CRISPR-Cas9 to replace disease-causing genetic variants with healthy DNA stretches, the Wall Street Journal reports.

It cautions that therapies based on such approaches won't be available for years, if at all, but adds that they offer "tremendous potential for new treatments."

"What if you could go right to the root cause of that disease and repair the broken gene? That's what people are excited about," Katrine Bosley, chief executive of Editas Medicine, says.

Her company, the Journal notes, is pursuing a CRISPR-Cas9-based therapeutic for Leber congenital amaurosis, while others are investigating its use to treat sickle-cell anemia, beta thalassemia, and blood cancers.

There are, though, a few stumbling blocks. There's a patent fight hanging over the field that involves the Broad Institute, the University of California, Umea University, and others regarding which team — Feng Zhang at the Broad or Berkeley's Jennifer Doudna and Umea's Emmanuelle Charpentier — first developed the approach.

There's also the question of how to get any CRISPR-Cas9-based therapy to its intended site. The first treatments will likely involve taking a sample of a patient's blood, treating the blood cells, and returning them, the Journal says, as other routes like using adeno-associated viruses or lipid nanoparticles are explored.

"In 10, 15 years, our relationship with genetic disease will be very different from today," Jacob Corn, managing director of the Innovative Genomics Initiative, tells the Journal. "It will be, 'Oh, my child was born with sickle cell. We're just going to change that.'"

The Scan

Positive Framing of Genetic Studies Can Spark Mistrust Among Underrepresented Groups

Researchers in Human Genetics and Genomics Advances report that how researchers describe genomic studies may alienate potential participants.

Small Study of Gene Editing to Treat Sickle Cell Disease

In a Novartis-sponsored study in the New England Journal of Medicine, researchers found that a CRISPR-Cas9-based treatment targeting promoters of genes encoding fetal hemoglobin could reduce disease symptoms.

Gut Microbiome Changes Appear in Infants Before They Develop Eczema, Study Finds

Researchers report in mSystems that infants experienced an enrichment in Clostridium sensu stricto 1 and Finegoldia and a depletion of Bacteroides before developing eczema.

Acute Myeloid Leukemia Treatment Specificity Enhanced With Stem Cell Editing

A study in Nature suggests epitope editing in donor stem cells prior to bone marrow transplants can stave off toxicity when targeting acute myeloid leukemia with immunotherapy.