gene therapy

To study the benefit of genetic testing data, the Foundation Fighting Blindness offered free clinical testing to 100 members of its My Retina Tracker patient registry.

NIH researchers got tantalizingly close to long-term engraftment at levels that could provide a clinical benefit in gene therapy applications.

The President's Council of Advisors on Science and Technology urges the adoption of a biodefense strategy, Tech Review reports.

A modified version of a commercially available magnetic bead cell sorting instrument from Miltenyi Biotec could be deployed to areas without cGMP facilities.

Some Cold Water

Even with all the current enthusiasm for gene therapies, Tech Review cautions that they are mostly in the early stages of development.

Scientists led by Eric Kmiec showed that their strategy of point mutation editing with single-stranded DNA oligos and double-strand breaks works with CRISPR/Cas9. 

Start It Themselves

Parents raise more than $1.8 million to start a clinical trial to help their daughter with Sanfilippo syndrome, Today reports.

Scientists introduced an indel to a promoter region to mimic a benign, naturally-occurring mutation that activated fetal hemoglobin production.

"Gene doping" may be the next method athletes use to give themselves an edge.

The pipeline, called IMPACT, pinpointed genetic variants in lung cancer and melanoma patients and matched them to targeted drugs. 

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Vivek Murthy is no longer the surgeon general of the US, the Associated Press reports.

People around the globe took to the streets to support science — some with signs.

Parents who learn of their increased genetic risk of disease also contend with telling their children about theirs, the New York Times writes.

In PLOS this week: loci linked to body mass index measurements, long non-coding RNA expression and urothelial carcinoma prognosis, and more.