People with Angelman syndrome face significant lifelong challenges. Scientists are developing three strategies to treat the disorder—relieve symptoms, reactivate the dormant UBE3A or apply traditional gene therapy.
Rare diseases are conditions affecting fewer than 200,000 people in the United States. The “overwhelming majority” of these diseases are far less prevalent, explained Dr. Anne Pariser, director of the Office of Rare Diseases Research at NCATS. In total, there are 25 million-30 million people with rare diseases in the United States.
The NIEHS cryo-electron microscopy facility, led by Dr. Mario Borgnia, is providing key support to the Duke Human Vaccine Institute in the fight against the SARS-CoV-2 virus, which produces COVID-19.
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A little-known ocean-dwelling creature, Hydractinia, most commonly found growing on dead hermit crab shells, may seem like an unlikely study subject for researchers, but this animal has a rare ability—it can make eggs and sperm for the duration of its lifetime. It produces germ cells—precursors to eggs and sperm—nonstop throughout its life. Studying this unique ability could provide insight into the formation of reproductive-based conditions and diseases in humans.