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Stem Cell Studies Underway

The US government has OK'd a human study using a therapy derived from embryonic stem cells to treat Stargardt disease, a rare disease that causes loss of vision, says the AP's Malcolm Ritter. This is only the second embryonic stem cell study the government has ever approved in humans. It will be conducted by California biotech company Advanced Cell Technology, and should begin early in 2011. Though Stargardt disease is rare, ACT hopes that the same approach, if successful, could also be used to treat more common eye problems like macular degeneration, Ritter says. Last month, Geron Corp. began preliminary testing in humans to treat spinal cord injuries with cells derived from embryonic stem cells, he adds.

The Scan

Study Finds Few FDA Post-Market Regulatory Actions Backed by Research, Public Assessments

A Yale University-led team examines in The BMJ safety signals from the US FDA Adverse Event Reporting System and whether they led to regulatory action.

Duke University Team Develops Programmable RNA Tool for Cell Editing

Researchers have developed an RNA-based editing tool that can target specific cells, as they describe in Nature.

Novel Gene Editing Approach for Treating Cystic Fibrosis

Researchers in Science Advances report on their development of a non-nuclease-based gene editing approach they hope to apply to treat cystic fibrosis.

Study Tracks Responses in Patients Pursuing Polygenic Risk Score Profiling

Using interviews, researchers in the European Journal of Human Genetics qualitatively assess individuals' motivations for, and experiences with, direct-to-consumer polygenic risk score testing.