Thalidomide’s harms, though well documented, have never been completely understood at the molecular level. Now, more than 60 years after thalidomide caused birth defects in thousands of children whose mothers took the drug while pregnant, scientists based at the Dana-Farber Cancer Institute have finally solved the molecular mystery. According to these scientists, thalidomide acts by promoting the degradation of a transcription factor called SALL4. Actually, thalidomide causes the degradation of a surprisingly wide range of transcription factors, which are cell proteins that help switch genes on or off. But SALL4 is of special interest because it is also implicated in Duane Ray Syndrome. Specifically, SALL4 is mutated in Duane Ray Syndrome, which is evident in newborns and is characterized by missing thumbs, underdeveloped limbs, eye and ear defects, and congenital heart disease—problems that mirror those in children exposed to thalidomide in the womb. The similarities between the ...
Original Article: Thalidomide’s Birth-Defect-Causing Mechanism Finally Uncovered