نتایج جستجو برای: smn gene

تعداد نتایج: 1142093  

Journal: :Human molecular genetics 1999
U R Monani C L Lorson D W Parsons T W Prior E J Androphy A H Burghes J D McPherson

Spinal muscular atrophy (SMA) is a recessive disorder characterized by loss of motor neurons in the spinal cord. It is caused by mutations in the telomeric survival motor neuron 1 ( SMN1 ) gene. Alterations within an almost identical copy gene, the centromeric survival motor neuron 2 ( SMN2 ) gene produce no known phenotypic effect. The exons of the two genes differ by just two nucleotides, nei...

Journal: :Human molecular genetics 2010
Christian M Simon Sibylle Jablonka Rocio Ruiz Lucia Tabares Michael Sendtner

Proximal spinal muscular atrophy (SMA) is caused by homozygous loss or mutation of the SMN1 gene on human chromosome 5. Depending on the levels of SMN protein produced from a second SMN gene (SMN2), different forms of the disease are distinguished. In patients with milder forms of the disease, type III or type IV SMA that normally reach adulthood, enlargement of motor units is regularly observe...

Journal: :The Journal of neuroscience : the official journal of the Society for Neuroscience 2009
Jason H Williams Rebecca C Schray Carlyn A Patterson Semira O Ayitey Melanie K Tallent Gordon J Lutz

Spinal muscular atrophy (SMA) is caused by homozygous mutation or deletion of the SMN1 gene encoding survival of motor neuron (SMN) protein, resulting in the selective loss of alpha-motor neurons. Humans typically have one or more copies of the SMN2 gene, the coding region of which is nearly identical to SMN1, except that a point mutation causes splicing out of exon 7 and production of a largel...

2015
Vicki L. McGovern Aurélie Massoni-Laporte Xueyong Wang Thanh T. Le Hao T. Le Christine E. Beattie Mark M. Rich Arthur H. M. Burghes Udai Pandey

Spinal muscular atrophy is caused by loss of the SMN1 gene and retention of SMN2. The SMN2 copy number inversely correlates with phenotypic severity and is a modifier of disease outcome. The SMN2 gene essentially differs from SMN1 by a single nucleotide in exon 7 that modulates the incorporation of exon 7 into the final SMN transcript. The majority of the SMN2 transcripts lack exon 7 and this l...

2014
Magali Cucchiarini Henning Madry Ernest F Terwilliger

The identification of new components implicated in the pathogenesis of osteoarthritis (OA) might improve our understanding of the disease process. Here, we investigated the levels of the survival of motor neuron (SMN) expression in OA cartilage considering the fundamental role of the SMN protein in cell survival and its involvement in other stress-associated pathologies. We report that SMN expr...

2016
Hannah K. Shorrock Thomas H. Gillingwater Euan MacDonald

Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disorder characterised by widespread loss of lower motor neurons from the spinal cord, leading to progressive weakness and muscle atrophy. SMA is largely caused by homozygous loss of the survival motor neuron (SMN) 1 gene, resulting in reduced levels of full-length SMN protein. Although no approved treatment is currently avai...

2017
Wei Li

Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disease with dysfunctional α-motor neurons in the anterior horn of the spinal cord. SMA is caused by loss (∼95% of SMA cases) or mutation (∼5% of SMA cases) of the survival motor neuron 1 gene SMN1. As the product of SMN1, SMN is a component of the SMN complex, and is also involved in the biosynthesis of the small nuclear rib...

Journal: :Science 2014
Nikolai A Naryshkin Marla Weetall Amal Dakka Jana Narasimhan Xin Zhao Zhihua Feng Karen K Y Ling Gary M Karp Hongyan Qi Matthew G Woll Guangming Chen Nanjing Zhang Vijayalakshmi Gabbeta Priya Vazirani Anuradha Bhattacharyya Bansri Furia Nicole Risher Josephine Sheedy Ronald Kong Jiyuan Ma Anthony Turpoff Chang-Sun Lee Xiaoyan Zhang Young-Choon Moon Panayiota Trifillis Ellen M Welch Joseph M Colacino John Babiak Neil G Almstead Stuart W Peltz Loren A Eng Karen S Chen Jesse L Mull Maureen S Lynes Lee L Rubin Paulo Fontoura Luca Santarelli Daniel Haehnke Kathleen D McCarthy Roland Schmucki Martin Ebeling Manaswini Sivaramakrishnan Chien-Ping Ko Sergey V Paushkin Hasane Ratni Irene Gerlach Anirvan Ghosh Friedrich Metzger

Spinal muscular atrophy (SMA) is a genetic disease caused by mutation or deletion of the survival of motor neuron 1 (SMN1) gene. A paralogous gene in humans, SMN2, produces low, insufficient levels of functional SMN protein due to alternative splicing that truncates the transcript. The decreased levels of SMN protein lead to progressive neuromuscular degeneration and high rates of mortality. Th...

Journal: :Human molecular genetics 1998
S Lefebvre L Bürglen J Frézal A Munnich J Melki

Childhood spinal muscular atrophy (SMA) is a common recessive autosomal disorder that results in degeneration of lower motor neurons. The identification of the disease gene, Survival of Motor Neuron (SMN), was a major advance in understanding the molecular basis underlying this devastating neuromuscular disease. This finding has greatly improved the genetic counselling of SMA families. Recently...

2017
Sota Iwatani Nur Imma Fatimah Harahap Dian Kesumapramudya Nurputra Shinya Tairaku Akemi Shono Daisuke Kurokawa Keiji Yamana Khin Kyae Mon Thwin Makiko Yoshida Masami Mizobuchi Tsubasa Koda Kazumichi Fujioka Mariko Taniguchi-Ikeda Hideto Yamada Ichiro Morioka Kazumoto Iijima Hisahide Nishio Noriyuki Nishimura

BACKGROUND Spinal muscular atrophy (SMA) is the most common genetic neurological disease leading to infant death. It is caused by loss of survival motor neuron (SMN) 1 gene and subsequent reduction of SMN protein in motor neurons. Because SMN is ubiquitously expressed and functionally linked to general RNA metabolism pathway, fibroblasts (FBs) are most widely used for the assessment of SMN expr...

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