نتایج جستجو برای: smn2 protein

تعداد نتایج: 1234838  

Journal: :JCI insight 2016
Mahlet B Abera Jingbo Xiao Jonathan Nofziger Steve Titus Noel Southall Wei Zheng Kasey E Moritz Marc Ferrer Jonathan J Cherry Elliot J Androphy Amy Wang Xin Xu Christopher Austin Kenneth H Fischbeck Juan J Marugan Barrington G Burnett

Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disease and one of the leading inherited causes of infant mortality. SMA results from insufficient levels of the survival motor neuron (SMN) protein, and studies in animal models of the disease have shown that increasing SMN protein levels ameliorates the disease phenotype. Our group previously identified and optimized a new ...

2014
Zubia Mumtaz Andrea Cutherell Afshan Bhatti

BACKGROUND To address it's persistently high maternal mortality rate of 276/100,000 live births, the government of Pakistan created a new cadre of community based midwives (CMW). One expectation is that CMWs will improve access to maternal health services for underserved women. Recent research shows the CMWs have largely failed to establish midwifery practices, because CMW's lack of skills, bot...

Journal: :The Journal of clinical investigation 2011
Faraz Farooq Francisco Abadía Molina Jeremiah Hadwen Duncan MacKenzie Luke Witherspoon Matthew Osmond Martin Holcik Alex MacKenzie

Spinal muscular atrophy (SMA) is an autosomal recessive neurodegenerative disease that is characterized by the loss of motor neurons, resulting in progressive muscle atrophy. It is caused by the loss of functional survival motor neuron (SMN) protein due to mutations or deletion in the SMN1 gene. A potential treatment strategy for SMA is to upregulate levels of SMN protein. Several agents that a...

2015
Ping Fang Liang Li Jian Zeng Wan-Jun Zhou Wei-Qing Wu Ze-Yan Zhong Ti-Zhen Yan Jian-Sheng Xie Jing Huang Li Lin Ying Zhao Xiang-Min Xu

BACKGROUND Spinal muscular atrophy (SMA) is caused by SMN1 dysfunction, and the copy number of SMN2 and NAIP can modify the phenotype of SMA. The aim of this study was to analyze the copy numbers and gene structures of SMA-related genes in Chinese SMA patients and unrelated healthy controls. METHODS Forty-two Chinese SMA patients and two hundred and twelve unrelated healthy Chinese individual...

Journal: :Dalton transactions 2014
Jérémy Chaignon Salah-Eddine Stiriba Francisco Lloret Consuelo Yuste Guillaume Pilet Laurent Bonneviot Belén Albela Isabel Castro

Clickable ligands like N,N'-bis((pyridin-2-yl)methyl)prop-2-yn-1-amine (L(1)) and N-((1-methyl-1H-imidazol-2-yl)methyl)-N-(pyridin-2-ylmethyl)prop-2-yn-1-amine (L(2)) have been used to synthesise a series of manganese(ii) complexes for grafting onto appropriate solid supports. These ligands mimic the 2-His-1-carboxylate facial chelation present in the active site of the manganese-dependent diox...

2016
Afrooz RASHNONEJAD Huseyin ONAY Tahir ATIK Ozlem ATAN SAHIN Sarenur GOKBEN Hasan TEKGUL Ferda OZKINAY

OBJECTIVE To describe 12 yr experience of molecular genetic diagnosis of Spinal Muscular Atrophy (SMA) in 460 cases of Turkish patients. MATERIALS & METHODS A retrospective analysis was performed on data from 460 cases, referred to Medical Genetics Laboratory, Ege University's Hospital, Izmir, Turkey, prediagnosed as SMA or with family history of SMA between 2003 and 2014. The PCR-restriction...

Journal: :Journal of neuromuscular diseases 2014
Duncan MacKenzie Fahad Shamim Kevin Mongeon Ankur Trivedi Alex MacKenzie Faraz Farooq

BACKGROUND Autosomal recessive spinal muscle atrophy (SMA) is characterized by the loss of α motor neurons resulting in progressive muscle loss and respiratory failure. SMA is one of the most common inherited causes of infant death with a carrier frequency of 1 in 50 and a calculated prevalence of about 1 in 11,000 live births in the US. The low amount of functional survival motor neuron (SMN) ...

2016
Marc-Olivier Deguise Justin G. Boyer Emily R. McFall Armin Yazdani Yves De Repentigny Rashmi Kothary

Motor neuron loss and neurogenic atrophy are hallmarks of spinal muscular atrophy (SMA), a leading genetic cause of infant deaths. Previous studies have focused on deciphering disease pathogenesis in motor neurons. However, a systematic evaluation of atrophy pathways in muscles is lacking. Here, we show that these pathways are differentially activated depending on severity of disease in two dif...

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