نتایج جستجو برای: survival motor neuron smn gene

تعداد نتایج: 1595688  

Journal: :The Journal of neuroscience : the official journal of the Society for Neuroscience 2006
Honglai Zhang Lei Xing Wilfried Rossoll Hynek Wichterle Robert H Singer Gary J Bassell

Spinal muscular atrophy (SMA), a progressive neurodegenerative disease affecting motor neurons, is caused by mutations or deletions of the SMN1 gene encoding the survival of motor neuron (SMN) protein. In immortalized non-neuronal cell lines, SMN has been shown to form a ribonucleoprotein (RNP) complex with Gemin proteins, which is essential for the assembly of small nuclear RNPs (snRNPs). An a...

Journal: :The Journal of clinical investigation 2007
Amy M Avila Barrington G Burnett Addis A Taye Francesca Gabanella Melanie A Knight Parvana Hartenstein Ziga Cizman Nicholas A Di Prospero Livio Pellizzoni Kenneth H Fischbeck Charlotte J Sumner

The inherited motor neuron disease spinal muscular atrophy (SMA) is caused by mutation of the telomeric survival motor neuron 1 (SMN1) gene with retention of the centromeric SMN2 gene. We sought to establish whether the potent and specific hydroxamic acid class of histone deacetylase (HDAC) inhibitors activates SMN2 gene expression in vivo and modulates the SMA disease phenotype when delivered ...

Journal: :Human molecular genetics 2010
Thomas M Wishart Jack P-W Huang Lyndsay M Murray Douglas J Lamont Chantal A Mutsaers Jenny Ross Pascal Geldsetzer Olaf Ansorge Kevin Talbot Simon H Parson Thomas H Gillingwater

Reduced expression of the survival motor neuron (SMN) gene causes the childhood motor neuron disease spinal muscular atrophy (SMA). Low levels of ubiquitously expressed SMN protein result in the degeneration of lower motor neurons, but it remains unclear whether other regions of the nervous system are also affected. Here we show that reduced levels of SMN lead to impaired perinatal brain develo...

2010
Gideon Dreyfuss Howard Hughes

Spinal muscular atrophy (SMA) is the most common motor neuron degenerative disease and is the principal genetic cause of infant mortality, affecting 1 in every 6000 newborns. The survival of motor neurons (SMN) gene has been implicated as the disease-causing gene in SMA, and it is deleted or mutated in over 98% of SMA patients. Our lab has pioneered research elucidating the functions of the SMN...

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 2012
Shingo Kariya Diane B Re Arnaud Jacquier Katelyn Nelson Serge Przedborski Umrao R Monani

Spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis (ALS) are among the most common motor neuron diseases to afflict the human population. A deficiency of the survival of motor neuron (SMN) protein causes SMA and is also reported to be an exacerbating factor in the development of ALS. However, pathways linking the two diseases have yet to be defined and it is not clear precisely how...

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...

Journal: :Human molecular genetics 2013
Le T Hao Phan Q Duy James D Jontes Marc Wolman Michael Granato Christine E Beattie

Proper function of the motor unit is dependent upon the correct development of dendrites and axons. The infant/childhood onset motoneuron disease spinal muscular atrophy (SMA), caused by low levels of the survival motor neuron (SMN) protein, is characterized by muscle denervation and paralysis. Although different SMA models have shown neuromuscular junction defects and/or motor axon defects, a ...

2009
Dirk Bäumer Sheena Lee George Nicholson Joanna L. Davies Nicholas J. Parkinson Lyndsay M. Murray Thomas H. Gillingwater Olaf Ansorge Kay E. Davies Kevin Talbot

Spinal muscular atrophy is a severe motor neuron disease caused by inactivating mutations in the SMN1 gene leading to reduced levels of full-length functional SMN protein. SMN is a critical mediator of spliceosomal protein assembly, and complete loss or drastic reduction in protein leads to loss of cell viability. However, the reason for selective motor neuron degeneration when SMN is reduced t...

Journal: :Human molecular genetics 2014
James N Sleigh Antón Barreiro-Iglesias Peter L Oliver Angeliki Biba Thomas Becker Kay E Davies Catherina G Becker Kevin Talbot

Spinal muscular atrophy (SMA) is characterized by the selective loss of spinal motor neurons owing to reduced levels of survival motor neuron (Smn) protein. In addition to its well-established role in assembling constituents of the spliceosome, diverse cellular functions have been proposed for Smn, but the reason why low levels of this widely expressed protein result in selective motor neuron p...

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