Oculodentodigital dysplasia (ODDD) is a dominantly inherited human disorder associated with different symptoms like craniofacial
anomalies, syndactyly and heart dysfunction. ODDD is caused by mutations in the
GJA1 gene encoding the gap junction protein connexin43 (Cx43). Here, we have characterized four Cx43 mutations (I31M, G138R, G143S
and H194P) after stable expression in HeLa cells. In patients, the I31M and G138R mutations showed all phenotypic characteristics
of ODDD, whereas G143S did not result in facial abnormalities and H194P mutated patients exhibited no syndactylies. In transfected
HeLa cells, these mutations led to lack of the P2 phosphorylation state of the Cx43 protein, complete inhibition of gap junctional
coupling measured by neurobiotin transfer and increased hemichannel activity. In addition, altered trafficking and delayed
degradation were found in these mutants by immunofluorescence and pulse-chase analyses. In G138R and G143S mutants, the increased
hemichannel activity correlated with an increased half-time of the Cx43 protein. However, the I31M mutated protein showed
no extended half-time. Thus, the increased hemichannel activity may be directly caused by an altered conformation of the mutated
channel forming protein. We hypothesize that increased hemichannel activity may aggravate the phenotypic abnormalities in
ODDD patients who are deficient in Cx43 gap junction channels.
Keywords Gap junction - Connexin - Cx43 - Hemichannel - Oculodentodigital dysplasia
Radoslaw Dobrowolski and Annette Sommershof contributed equally to this work.