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外显子组测序揭示独立的 SGCD 缺失导致波士顿梗犬肢带型肌营养不良

Exome sequencing reveals independent SGCD deletions causing limb girdle muscular dystrophy in Boston terriers.

机构信息

CAG GmbH - Center for Animal Genetics, Paul-Ehrlich-Str. 23, 72076, Tubingen, Germany.

Department of Genetics and Biochemistry, Clemson University, 130 McGinty Ct., Clemson, SC, 29634, USA.

出版信息

Skelet Muscle. 2017 Jul 11;7(1):15. doi: 10.1186/s13395-017-0131-0.

Abstract

BACKGROUND

Limb-girdle muscular dystrophies (LGMDs) are a heterogeneous group of inherited autosomal myopathies that preferentially affect voluntary muscles of the shoulders and hips. LGMD has been clinically described in several breeds of dogs, but the responsible mutations are unknown. The clinical presentation in dogs is characterized by marked muscle weakness and atrophy in the shoulder and hips during puppyhood.

METHODS

Following clinical evaluation, the identification of the dystrophic histological phenotype on muscle histology, and demonstration of the absence of sarcoglycan-sarcospan complex by immunostaining, whole exome sequencing was performed on five Boston terriers: one affected dog and its three family members and one unrelated affected dog.

RESULTS

Within sarcoglycan-δ (SGCD), a two base pair deletion segregating with LGMD in the family was discovered, and a deletion encompassing exons 7 and 8 was found in the unrelated dog. Both mutations are predicted to cause an absence of SGCD protein, confirmed by immunohistochemistry. The mutations are private to each family.

CONCLUSIONS

Here, we describe the first cases of canine LGMD characterized at the molecular level with the classification of LGMD2F.

摘要

背景

肢带型肌营养不良症(LGMDs)是一组异质性遗传性常染色体肌病,主要影响肩部和臀部的随意肌。LGMD 已在几种犬种中进行了临床描述,但负责的突变尚不清楚。犬的临床表现特征是在幼犬期肩部和臀部出现明显的肌肉无力和萎缩。

方法

在进行临床评估后,通过肌肉组织学上的营养不良组织学表型的鉴定,以及免疫染色显示肌聚糖-肌联蛋白复合物缺失,对五只波士顿梗犬进行了全外显子组测序:一只患病犬及其三只家族成员和一只无关的患病犬。

结果

在肌聚糖-δ(SGCD)中,发现了与家族性 LGMD 分离的两个碱基对缺失,并在无关犬中发现了包含外显子 7 和 8 的缺失。这两种突变都预计会导致 SGCD 蛋白缺失,免疫组化证实了这一点。这些突变是每个家族特有的。

结论

在这里,我们描述了第一个在分子水平上具有 LGMD2F 分类特征的犬 LGMD 病例。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/5266/5506588/ac74049c313e/13395_2017_131_Fig1_HTML.jpg

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