gene therapy | GenomeWeb

gene therapy

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. 

For several reasons, scientists, as well as gene editing therapy company Editas, think that the first clinical trials for CRISPR-based cures will be for hereditary eye diseases.

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A Karmagenes researcher has lost his position after reportedly admitting to data fabrication, according to Retraction Watch.

Two neuroscientists write in Nature News that solving the "reproducibility crisis" in science may require changing the requirements for publication.

In Nature this week: genomic analysis of prehistoric New Mexicans, a nanopore method for mapping DNA methylation, and more.

A new study finds that adding missing good bacteria to the skin microbiome of atopic dermatitis patients decreases Staphylococcus aureus colonization.