Loss-of-function mutations in IGSF1 cause an X-linked syndrome of central hypothyroidism and testicular enlargement

Nat Genet. 2012 Dec;44(12):1375-81. doi: 10.1038/ng.2453. Epub 2012 Nov 11.

Abstract

Congenital central hypothyroidism occurs either in isolation or in conjunction with other pituitary hormone deficits. Using exome and candidate gene sequencing, we identified 8 distinct mutations and 2 deletions in IGSF1 in males from 11 unrelated families with central hypothyroidism, testicular enlargement and variably low prolactin concentrations. IGSF1 is a membrane glycoprotein that is highly expressed in the anterior pituitary gland, and the identified mutations impair its trafficking to the cell surface in heterologous cells. Igsf1-deficient male mice show diminished pituitary and serum thyroid-stimulating hormone (TSH) concentrations, reduced pituitary thyrotropin-releasing hormone (TRH) receptor expression, decreased triiodothyronine concentrations and increased body mass. Collectively, our observations delineate a new X-linked disorder in which loss-of-function mutations in IGSF1 cause central hypothyroidism, likely secondary to an associated impairment in pituitary TRH signaling.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Animals
  • Base Sequence
  • Child
  • Child, Preschool
  • Congenital Hypothyroidism / genetics*
  • Exome
  • Ferrous Compounds
  • Genetic Diseases, X-Linked / genetics*
  • Humans
  • Immunoglobulins / genetics*
  • Infant
  • Male
  • Membrane Proteins / genetics*
  • Metallocenes
  • Mice
  • Middle Aged
  • Mutation*
  • Pituitary Gland / metabolism
  • Pituitary Gland / pathology
  • Prolactin / blood
  • Receptors, Thyrotropin-Releasing Hormone / biosynthesis
  • Sequence Analysis, DNA
  • Syndrome
  • Testicular Diseases / genetics*
  • Testis / anatomy & histology
  • Testis / pathology
  • Thyrotropin / blood
  • Triiodothyronine / analysis
  • Young Adult

Substances

  • Ferrous Compounds
  • IGSF1 protein, human
  • Immunoglobulins
  • Membrane Proteins
  • Metallocenes
  • Receptors, Thyrotropin-Releasing Hormone
  • Triiodothyronine
  • EX 10-478
  • Prolactin
  • Thyrotropin