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gene therapy

Nature News reports that gene therapy approaches are tackling sickle cell disease, but that the cost of treatment is a concern.

Pause Button Hit

Novartis has paused a trial of its Zolgensma gene therapy that treats spinal muscular atrophy, the Wall Street Journal reports.

Health insurers in the US are devising new approaches to handle the high costs of gene therapies, the Wall Street Journal reports.

Novartis replaced two top executives from its AveXis unit following data manipulation during early testing of its new gene therapy, Reuters reports.

Data, Manipulated

US Food and Drug Administration says some data from early testing of Novartis' Zolgensma was manipulated, according to Reuters.

Slow Adoption

Reuters reports that slow adoption of CAR-T therapies might be tied to their high prices.

The company, which spun out of the Netherlands Cancer Institute, has developed technology to identify promoters and enhancers.

For Coverage

Parents of children with spinal muscular atrophy tell the Washington Post they are pushing to get insurance coverage of Novartis's Zolgensma.

Priciest Yet

Novartis's newly approved Zolgensma, a gene therapy for spinal muscular atrophy, is to cost $2.1 million, the AP says.

Looking Into It

A second death in gene therapy trial for type 1 spinal muscular atrophy is under investigation, according to Reuters.

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Forensic genetic firm Verogen has bought the genetic genealogy site GEDmatch.

Researchers have 3D-printed plastic bunnies that encase the information needed to make more such bunnies in DNA, according to Discover magazine.

Dan Rather, the former CBS Evening News anchor and executive producer of a new documentary, writes at the Guardian that everyone needs to know about CRISPR.

In PNAS this week: analysis of FOXA1 upregulation in ER-positive breast cancer, gene editing to correct recessive dystrophic epidermolysis bullosa, and more.