Northumbria Healthcare Trust, North Shields NE29 8NH, UK.
Newcastle University Medical School, Newcastle NE2 4HH, UK.
Int J Mol Sci. 2023 Aug 31;24(17):13545. doi: 10.3390/ijms241713545.
Congenital arthrogryposis (CA) refers to the presence of multiple contractures at birth. It is a feature of several inherited syndromes, notable amongst them are disorders of collagen formation. This review aims to characterize disorders that directly or indirectly impact collagen structure and function leading to CA in search for common phenotypic or pathophysiological features, possible genotype-phenotype correlation, and potential novel treatment approaches based on a better understanding of the underlying pathomechanism. Nine genes, corresponding to five clinical phenotypes, were identified after a literature search. The most notable trend was the extreme phenotype variability. Clinical features across all syndromes ranged from subtle with minimal congenital contractures, to severe with multiple congenital contractures and extra-articular features including skin, respiratory, or other manifestations. Five of the identified genes were involved in the function of the Lysyl Hydroxylase 2 or 3 enzymes, which enable the hydroxylation and/or glycosylation of lysyl residues to allow the formation of the collagen superstructure. Whilst current treatment approaches are post-natal surgical correction, there are also potential in-utero therapies being developed. Cyclosporin A showed promise in treating collagen VI disorders although there is an associated risk of immunosuppression. The treatments that could be in the clinical trials soon are the splice correction therapies in collagen VI-related disorders.
先天性关节挛缩症(CA)是指出生时存在多处挛缩。它是几种遗传性综合征的特征之一,其中值得注意的是胶原形成障碍。本综述旨在描述直接或间接影响胶原结构和功能导致 CA 的疾病,以寻找常见的表型或病理生理学特征、可能的基因型-表型相关性,以及基于对潜在发病机制的更好理解的潜在新治疗方法。在文献检索后,确定了九个与五个临床表型相对应的基因。最显著的趋势是表型极端变异性。所有综合征的临床特征从轻微的先天性挛缩到严重的多发性先天性挛缩以及关节外特征,包括皮肤、呼吸或其他表现。五个已确定的基因参与赖氨酰羟化酶 2 或 3 酶的功能,该酶使赖氨酸残基羟化和/或糖基化,从而形成胶原超结构。虽然目前的治疗方法是产后手术矫正,但也在开发潜在的宫内治疗方法。环孢素 A 在治疗胶原 VI 疾病方面显示出前景,尽管存在免疫抑制的相关风险。很快可能进入临床试验的治疗方法是胶原 VI 相关疾病的剪接纠正治疗。