scholarly journals A Mosaic Mutation in the LAMA2 Gene in a Case of Merosin-deficient Congenital Muscular Dystrophy

2021 ◽  
Vol 12 ◽  
Author(s):  
P. A. Chausova ◽  
O. P. Ryzhkova ◽  
G. E. Rudenskaya ◽  
V. B. Chernykh ◽  
O. A. Shchagina ◽  
...  

Merosine deficient congenital muscular dystrophy is one of the most common forms of congenital muscular dystrophy. This disease is caused by a primary deficiency or a functionally inactive form of the protein merosin in muscle tissue. The type of inheritance of this disease is autosomal recessive. De novo variants with this type of inheritance are rare, and it is quite possible that the de novo variant may hide a mosaic form in the parent of an affected child. We present a birth family with two affected children who inherited a previously undescribed pathogenic variant c.1755del from their mother and a previously described pathogenic variant c.9253C > T in the LAMA2 gene from their mosaic father. LAMA2 gene mutation analysis was performed by mass parallel sequencing and direct sequencing of genomic DNAs.

2020 ◽  
Vol 7 (1) ◽  
pp. 69-76
Author(s):  
Rocío N. Villar-Quiles ◽  
Marta Gomez-Garcia de la Banda ◽  
Annie Barois ◽  
Celine Bouchet-Séraphin ◽  
Norma B. Romero ◽  
...  

2017 ◽  
Vol 39 (4) ◽  
pp. 361-364 ◽  
Author(s):  
Pakize Karaoglu ◽  
Nicolas Quizon ◽  
Matthias Pergande ◽  
Haicui Wang ◽  
Ayşe Ipek Polat ◽  
...  

2019 ◽  
Vol 29 ◽  
pp. S168-S169
Author(s):  
T. Sato ◽  
N. Taniguchi ◽  
K. Ishiguro ◽  
M. Shichiji ◽  
T. Murakami ◽  
...  

2021 ◽  
Vol 22 (8) ◽  
pp. 4256
Author(s):  
Lorenzo Maggi ◽  
Manolis Mavroidis ◽  
Stelios Psarras ◽  
Yassemi Capetanaki ◽  
Giovanna Lattanzi

Intermediate filaments are major components of the cytoskeleton. Desmin and synemin, cytoplasmic intermediate filament proteins and A-type lamins, nuclear intermediate filament proteins, play key roles in skeletal and cardiac muscle. Desmin, encoded by the DES gene (OMIM *125660) and A-type lamins by the LMNA gene (OMIM *150330), have been involved in striated muscle disorders. Diseases include desmin-related myopathy and cardiomyopathy (desminopathy), which can be manifested with dilated, restrictive, hypertrophic, arrhythmogenic, or even left ventricular non-compaction cardiomyopathy, Emery–Dreifuss Muscular Dystrophy (EDMD2 and EDMD3, due to LMNA mutations), LMNA-related congenital Muscular Dystrophy (L-CMD) and LMNA-linked dilated cardiomyopathy with conduction system defects (CMD1A). Recently, mutations in synemin (SYNM gene, OMIM *606087) have been linked to cardiomyopathy. This review will summarize clinical and molecular aspects of desmin-, lamin- and synemin-related striated muscle disorders with focus on LMNA and DES-associated clinical entities and will suggest pathogenetic hypotheses based on the interplay of desmin and lamin A/C. In healthy muscle, such interplay is responsible for the involvement of this network in mechanosignaling, nuclear positioning and mitochondrial homeostasis, while in disease it is disturbed, leading to myocyte death and activation of inflammation and the associated secretome alterations.


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