Mutations affecting the cytoplasmic functions of the co-chaperone DNAJB6 cause limb-girdle muscular dystrophy.

Journal Article (Journal Article)

Limb-girdle muscular dystrophy type 1D (LGMD1D) was linked to chromosome 7q36 over a decade ago, but its genetic cause has remained elusive. Here we studied nine LGMD-affected families from Finland, the United States and Italy and identified four dominant missense mutations leading to p.Phe93Leu or p.Phe89Ile changes in the ubiquitously expressed co-chaperone DNAJB6. Functional testing in vivo showed that the mutations have a dominant toxic effect mediated specifically by the cytoplasmic isoform of DNAJB6. In vitro studies demonstrated that the mutations increase the half-life of DNAJB6, extending this effect to the wild-type protein, and reduce its protective anti-aggregation effect. Further, we show that DNAJB6 interacts with members of the CASA complex, including the myofibrillar myopathy-causing protein BAG3. Our data identify the genetic cause of LGMD1D, suggest that its pathogenesis is mediated by defective chaperone function and highlight how mutations in a ubiquitously expressed gene can exert effects in a tissue-, isoform- and cellular compartment-specific manner.

Full Text

Duke Authors

Cited Authors

  • Sarparanta, J; Jonson, PH; Golzio, C; Sandell, S; Luque, H; Screen, M; McDonald, K; Stajich, JM; Mahjneh, I; Vihola, A; Raheem, O; Penttilä, S; Lehtinen, S; Huovinen, S; Palmio, J; Tasca, G; Ricci, E; Hackman, P; Hauser, M; Katsanis, N; Udd, B

Published Date

  • February 26, 2012

Published In

Volume / Issue

  • 44 / 4

Start / End Page

  • 450 - S2

PubMed ID

  • 22366786

Pubmed Central ID

  • PMC3315599

Electronic International Standard Serial Number (EISSN)

  • 1546-1718

Digital Object Identifier (DOI)

  • 10.1038/ng.1103

Language

  • eng

Conference Location

  • United States