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一种新型肌营养不良蛋白(DMD)基因突变导致的猫 X 连锁肌营养不良症与轻度临床病程相关。

Association of a novel dystrophin (DMD) genetic nonsense variant in a cat with X-linked muscular dystrophy with a mild clinical course.

机构信息

Otakibashi Animal Hospital, Tokyo, Japan.

Laboratory of Veterinary Radiology, Nippon Veterinary and Life Science University, Tokyo, Japan.

出版信息

J Vet Intern Med. 2024 Mar-Apr;38(2):1160-1166. doi: 10.1111/jvim.17024. Epub 2024 Feb 28.

Abstract

X-linked muscular dystrophy in cats (FXMD) is an uncommon disease, with few reports describing its pathogenic genetic variants. A 9-year-old castrated male domestic shorthair cat was presented with persistent muscle swelling and breathing difficulty from 3 years of age. Serum activity of alanine aminotransferase, aspartate transaminase, and creatine kinase were abnormally high. Physical and neurological examinations showed muscle swelling in the neck and proximal limb, slow gait, and occasional breathing difficulties. Electromyography showed pseudomyotonic discharges and complex repetitive discharges with a "dive-bomber" sound. Histopathology revealed muscle necrosis and regeneration. Whole-genome sequencing identified a novel and unique hemizygous nonsense genetic variant, c.8333G > A in dystrophin (DMD), potentially causing a premature termination codon (p.Trp2778Ter). Based on a combination of clinical and histological findings and the presence of the DMD nonsense genetic variant, this case was considered FXMD, which showed mild clinical signs and long-term survival, even though immunohistochemical characterization was lacking.

摘要

猫 X 连锁肌营养不良症(FXMD)是一种罕见疾病,仅有少数报道描述了其致病的遗传变异。一只 9 岁已去势雄性短毛家猫,从 3 岁开始出现持续性肌肉肿胀和呼吸困难。血清丙氨酸氨基转移酶、天冬氨酸氨基转移酶和肌酸激酶活性异常升高。体格检查和神经学检查显示颈部和近端肢体肌肉肿胀、步态缓慢和偶尔呼吸困难。肌电图显示假性肌强直放电和复杂重复放电,伴有“俯冲轰炸机”样声音。组织病理学显示肌肉坏死和再生。全基因组测序发现一种新型独特的半合子无义遗传变异 c.8333G>A 位于肌营养不良蛋白(DMD)中,可能导致提前终止密码子(p.Trp2778Ter)。根据临床和组织学发现以及 DMD 无义遗传变异的存在,本例被认为是 FXMD,尽管缺乏免疫组织化学特征,但表现为轻度临床症状和长期存活。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b76b/10937502/ea2d0860ee4a/JVIM-38-1160-g003.jpg

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