Asubio Pharma Co., Ltd. Kobe, Japan.
Daiichi Sankyo Co., Ltd. Tokyo, Japan.
PLoS One. 2019 May 23;14(5):e0216340. doi: 10.1371/journal.pone.0216340. eCollection 2019.
C-type natriuretic peptide (CNP)-knockout (KO) rats exhibit impaired skeletal growth, with long bones shorter than those in wild-type (WT) rats. This study compared craniofacial morphology in the CNP-KO rat with that in the Spontaneous Dwarf Rat (SDR), a growth hormone (GH)-deficient model. The effects of subcutaneous administration of human CNP with 53 amino acids (CNP-53) from 5 weeks of age for 4 weeks on craniofacial morphology in CNP-KO rats were also investigated. Skulls of CNP-KO rats at 9 weeks of age were longitudinally shorter and the foramen magnum was smaller than WT rats. There were no differences in foramen magnum stenosis and midface hypoplasia between CNP-KO rats at 9 and 33 weeks of age. These morphological features were the same as those observed in CNP-KO mice and activated fibroblast growth factor receptor 3 achondroplasia-phenotype mice. In contrast, SDR did not exhibit foramen magnum stenosis and midface hypoplasia, despite shorter stature than in control rats. After administration of exogenous CNP-53, the longitudinal skull length and foramen magnum size in CNP-KO rats were significantly greater, and full or partial rescue was confirmed. The synchondrosis at the cranial base in CNP-KO rats is closed at 9 weeks, but not at 4 weeks of age. In contrast, synchondrosis closure in CNP-KO rats treated with CNP-53 was incomplete at 9 weeks of age. Administration of exogenous CNP-53 accelerated craniofacial skeletogenesis, leading to improvement in craniofacial morphology. As these findings in CNP-KO rats are similar to those in patients with achondroplasia, treatment with CNP-53 or a CNP analog may be able to restore craniofacial morphology and foramen magnum size as well as short stature.
C 型利钠肽(CNP)敲除(KO)大鼠表现出骨骼生长受损,长骨比野生型(WT)大鼠短。本研究比较了 CNP-KO 大鼠与生长激素(GH)缺乏模型自发性矮小大鼠(SDR)的颅面形态。还研究了从 5 周龄开始,每周皮下给予 53 个氨基酸的人 CNP(CNP-53)4 周对 CNP-KO 大鼠颅面形态的影响。9 周龄 CNP-KO 大鼠的颅骨纵向较短,枕骨大孔较小。9 周和 33 周龄 CNP-KO 大鼠的枕骨大孔狭窄和中面部发育不全无差异。这些形态特征与 CNP-KO 小鼠和激活成纤维细胞生长因子受体 3 软骨发育不全表型小鼠观察到的特征相同。相比之下,尽管 SDR 比对照大鼠矮,但没有出现枕骨大孔狭窄和中面部发育不全。给予外源性 CNP-53 后,CNP-KO 大鼠的颅长和枕骨大孔大小显著增加,并证实完全或部分恢复。CNP-KO 大鼠颅底的骺板在 9 周时闭合,但在 4 周时没有闭合。相反,用 CNP-53 治疗的 CNP-KO 大鼠的骺板闭合在 9 周时不完全。给予外源性 CNP-53 可加速颅面骨骼生成,从而改善颅面形态。由于 CNP-KO 大鼠的这些发现与软骨发育不全患者的相似,因此 CNP-53 或 CNP 类似物的治疗可能能够恢复颅面形态和枕骨大孔大小以及身材矮小。