Bicknell L S, Morgan T, Bonafé L, Wessels M W, Bialer M G, Willems P J, Cohn D H, Krakow D, Robertson S P
J Med Genet. 2005 Jul;42(7):e43. doi: 10.1136/jmg.2004.029967.
Boomerang dysplasia (BD) is a perinatal lethal osteochondrodysplasia, characterised by absence or underossification of the limb bones and vertebrae. The BD phenotype is similar to a group of disorders including atelosteogenesis I, atelosteogenesis III, and dominantly inherited Larsen syndrome that we have recently shown to be associated with mutations in FLNB, the gene encoding the actin binding cytoskeletal protein, filamin B. We report the identification of mutations in FLNB in two unrelated individuals with boomerang dysplasia. The resultant substitutions, L171R and S235P, lie within the calponin homology 2 region of the actin binding domain of filamin B and occur at sites that are evolutionarily well conserved. These findings expand the phenotypic spectrum resulting from mutations in FLNB and underline the central role this protein plays during skeletogenesis in humans.
回飞镖发育异常(BD)是一种围产期致死性骨软骨发育不良,其特征为四肢骨骼和椎骨缺失或骨化不全。BD的表型与一组疾病相似,包括I型atelosteogenesis、III型atelosteogenesis和显性遗传的拉森综合征,我们最近发现这些疾病与编码肌动蛋白结合细胞骨架蛋白细丝蛋白B的基因FLNB中的突变有关。我们报告了在两名患有回飞镖发育异常的无关个体中鉴定出FLNB中的突变。产生的替代突变L171R和S235P位于细丝蛋白B肌动蛋白结合域的钙调蛋白同源2区域内,且发生在进化上高度保守的位点。这些发现扩展了由FLNB突变导致的表型谱,并强调了该蛋白在人类骨骼发育过程中所起的核心作用。