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Article

Rapamycin for treating Tuberous sclerosis and Autism spectrum disorders.

Trends in molecular medicine - 1 Feb 2011

Ehninger Dan, Silva Alcino J

Abstract excerpt

Tuberous sclerosis (TSC) is a genetic disorder caused by heterozygous mutations in the TSC1 or TSC2 genes and is associated with autism spectrum disorders (ASD) in 20-60% of cases. In addition, altered TSC/mTOR signaling is emerging as a feature common to a subset of ASD. Recent findings, in animal models, show that restoration of the underlying molecular defect can improve neurological dysfunction in several of...

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Source

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Identifiers

PMID: 21115397
DOI: 10.1016/j.molmed.2010.10.002
PMCID: PMC3075964

Genes

TSC1
TSC2

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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