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.

Pages

In Science this week: metagenomic-based technique for determining protein structure, and more.

An academic laments the rise of narcissism in the sciences, the Guardian reports.

Outgoing FDA commissioner Robert Califf writes in an editorial that the agency can help boost innovation.

The Trump transition team has asked NIH Director Francis Collins to remain at his post, though it's unclear for how long that will be.