Clinical and molecular insights into tuberous sclerosis complex renal disease

Pediatr Nephrol. 2011 Jun;26(6):839-52. doi: 10.1007/s00467-010-1689-5. Epub 2010 Dec 9.

Abstract

Patients with tuberous sclerosis complex are at great risk of developing renal lesions as part of their disease. These lesions include renal cysts and tumors. Significant advances in understanding the cell biology of these renal lesions has already led to clinical trials demonstrating that pharmacological interventions are likely possible. This review focuses on the pathology of these renal lesions, their underlying cell biology, and the possible therapeutic strategies that may prove to significantly improve care for these patients.

Publication types

  • Research Support, U.S. Gov't, Non-P.H.S.
  • Review

MeSH terms

  • Angiomyolipoma / drug therapy
  • Angiomyolipoma / etiology*
  • Angiomyolipoma / pathology
  • Antibiotics, Antineoplastic / therapeutic use
  • Child
  • Humans
  • Kidney Diseases, Cystic / drug therapy
  • Kidney Diseases, Cystic / etiology*
  • Kidney Diseases, Cystic / pathology
  • Kidney Neoplasms / drug therapy
  • Kidney Neoplasms / etiology*
  • Kidney Neoplasms / pathology
  • Mechanistic Target of Rapamycin Complex 1
  • Multiprotein Complexes
  • Proteins / physiology
  • Sirolimus / therapeutic use
  • TOR Serine-Threonine Kinases
  • Tuberous Sclerosis / complications*
  • Tuberous Sclerosis / drug therapy
  • Tuberous Sclerosis / pathology

Substances

  • Antibiotics, Antineoplastic
  • Multiprotein Complexes
  • Proteins
  • Mechanistic Target of Rapamycin Complex 1
  • TOR Serine-Threonine Kinases
  • Sirolimus