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Genetic Linkage

From Blue Lights to Gene Therapy: The Intriguing History of Crigler-Najjar Syndrome

Seeing Crigler-Najjar syndrome among recent news releases announcing upcoming gene therapy efforts conjured immediate images of an Amish farmhouse with a spooky blue glow emanating from an upstairs bedroom, where a small child, clad only in diapers and wearing goggles, slept beneath a “light canopy” suspended from the ceiling. The light – phototherapy or “bili lights” – breaks down the accumulating bilirubin that the tiny yellow body beneath cannot.

Most cases of neonatal jaundice are due to a transient blood type incompatability or blocked bile ducts, and vanish within days. Not so

Crigler-Najjar syndrome, which hampers the ability to convert the bile pigment bilirubin into a soluble form that easily exits in feces. Read More 
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