Pathogenic variants of the <i>GNAS</i> gene introduce an abnormal amino acid sequence in the β6 strand/α5 helix of Gsα, causing pseudohypoparathyroidism type 1A and pseudopseudohypoparathyroidism in two unrelated Japanese families.

Ohata, Yasuhisa; Kakimoto, Haruna; Seki, Yuko; Ishihara, Yasuki; Nakano, Yukako; Yamamoto, Kenichi; Takeyari, Shinji; Fujiwara, Makoto et al. · Bone Rep · 2022

basic_science · Level V

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Abstract

Pseudohypoparathyroidism 1A (PHP1A) and pseudopseudohypoparathyroidism (PPHP) are caused by loss-of-function variants of <i>GNAS</i>, which encodes Gsα. We present two unrelated Japanese families with PHP1A and PPHP harboring unreported pathogenic variants of <i>GNAS</i> (c.1141delG, p.Asp381Thrfs*23 and c.1117delC, p.Arg373Alafs*31). These variants introduce abnormal amino acids in the β6 strand/α5 helix of Gsα, which interact with G protein coupling receptor (GPCR). We conclude that these variants alter the association of Gsα with GPCR and cause PHP1A or PPHP.