NHERF1 mutations and responsiveness of renal parathyroid hormone

N Engl J Med. 2008 Sep 11;359(11):1128-35. doi: 10.1056/NEJMoa0802836.

Abstract

Impaired renal phosphate reabsorption, as measured by dividing the tubular maximal reabsorption of phosphate by the glomerular filtration rate (TmP/GFR), increases the risks of nephrolithiasis and bone demineralization. Data from animal models suggest that sodium-hydrogen exchanger regulatory factor 1 (NHERF1) controls renal phosphate transport. We sequenced the NHERF1 gene in 158 patients, 94 of whom had either nephrolithiasis or bone demineralization. We identified three distinct mutations in seven patients with a low TmP/GFR value. No patients with normal TmP/GFR values had mutations. The mutants expressed in cultured renal cells increased the generation of cyclic AMP (cAMP) by parathyroid hormone (PTH) and inhibited phosphate transport. These NHERF1 mutations suggest a previously unrecognized cause of renal phosphate loss in humans.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Animals
  • Biological Transport / genetics
  • Bone Demineralization, Pathologic / genetics*
  • Bone Demineralization, Pathologic / metabolism
  • Bone Demineralization, Pathologic / physiopathology
  • Cells, Cultured
  • Cyclic AMP / biosynthesis
  • Cyclic AMP / urine
  • DNA Mutational Analysis
  • Female
  • Glomerular Filtration Rate / genetics
  • Humans
  • Hypercalciuria / genetics
  • Kidney / cytology
  • Kidney / metabolism
  • Kidney Calculi / genetics*
  • Kidney Calculi / metabolism
  • Kidney Calculi / physiopathology
  • Male
  • Middle Aged
  • Mutation
  • Mutation, Missense
  • Nephrolithiasis / genetics*
  • Nephrolithiasis / metabolism
  • Opossums
  • Parathyroid Hormone / blood
  • Parathyroid Hormone / metabolism*
  • Phosphates / blood
  • Phosphates / metabolism*
  • Phosphoproteins / genetics*
  • Sodium-Hydrogen Exchangers / genetics*

Substances

  • Parathyroid Hormone
  • Phosphates
  • Phosphoproteins
  • Sodium-Hydrogen Exchangers
  • sodium-hydrogen exchanger regulatory factor
  • Cyclic AMP