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Mutagenesis

Rapamycin and similar agents might treat tuberous sclerosis complex

Published in Gene Therapy Weekly, November 13th, 2003

Rapamycin and similar agents might treat tuberous sclerosis complex.

"Mutation in either TSC1 or TSC2 causes the autosomal dominant disorder tuberous sclerosis, in which widespread hamartomas are seen, some of which have a high level of vascularization. Tuberous sclerosis complex (TSC) gene products negatively regulate mammalian target of rapamycin (mTOR) activity. We found that vascular endothelial growth factor (VEGF) is secreted by Tsc1- or Tsc2-null fibroblasts at high levels compared with wild-type cells," according to researchers in the Unite States.

"In Tsc1(+/-) mice, serum levels of VEGF were increased and appeared to be associated with the...

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