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gene therapy

GenomeWeb asked a number of prominent researchers in the field via email about their top picks for notable developments or achievements in genomics during the 2010s.

Walking Around

A Minnesota toddler given a gene therapy to treat her spinal muscular atrophy is now walking, according to Newsweek.

Therapy Lottery

The Guardian reports that parents of children with spinal muscular atrophy are concerned about a plan to have a lottery for free gene therapy doses.

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.

Pages

New analyses indicate female researchers are publishing less during the coronavirus pandemic than male researchers, according to Nature News.

A study suggests people with the ApoE e4 genotype may be more likely to have severe COVID-19 than those with other genotypes, the Guardian says.

Direct-to-consumer genetic testing companies are searching for a genetic reason for why some people, but not others, become gravely ill with COVID-19, the Detroit Free Press reports.

In PNAS this week: forward genetics-base analysis of retinal development, interactions of T cell receptors with neoantigens in colorectal cancer, and more.