Literature DB >> 29518354

Augmenting the SMN Protein to Treat Infantile Spinal Muscular Atrophy.

Jeong-Ki Kim1, Umrao R Monani2.   

Abstract

Spinal muscular atrophy (SMA) is a common and oft-fatal pediatric neuromuscular disorder caused by insufficient SMN protein. Now, two clinical trials (Mendell et al., 2017; Finkel et al., 2017) demonstrate that restoring the protein is therapeutic, offering new treatment options and renewed hope to SMA patients.
Copyright © 2018 Elsevier Inc. All rights reserved.

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Year:  2018        PMID： 29518354     DOI： 10.1016/j.neuron.2018.02.009

Source DB:  PubMed          Journal:  Neuron        ISSN： 0896-6273            Impact factor:   17.173


  4 in total

1.  Muscle-specific SMN reduction reveals motor neuron-independent disease in spinal muscular atrophy models.

Authors:  Jeong-Ki Kim; Narendra N Jha; Zhihua Feng; Michelle R Faleiro; Claudia A Chiriboga; Lan Wei-Lapierre; Robert T Dirksen; Chien-Ping Ko; Umrao R Monani
Journal:  J Clin Invest       Date:  2020-03-02       Impact factor: 14.808

2.  Motor neuron biology and disease: A current perspective on infantile-onset spinal muscular atrophy.

Authors:  Narendra N Jha; Jeong-Ki Kim; Umrao R Monani
Journal:  Future Neurol       Date:  2018-07-06

3.  Hyper-SUMOylation of SMN induced by SENP2 deficiency decreases its stability and leads to spinal muscular atrophy-like pathology.

Authors:  Yuhong Zhang; Xu Chen; Qiqi Wang; Congcong Du; Wenbin Lu; Hong Yuan; Zhenzhen Zhang; Danqing Li; Xing Ling; Xiang Ren; Yang Zhao; Qi Su; Zhengcao Xing; Yuanyuan Qin; Xinyi Yang; Yajie Shen; Hongmei Wu; Yitao Qi
Journal:  J Mol Med (Berl)       Date:  2021-10-09       Impact factor: 4.599

Review 4.  Novel genome-editing-based approaches to treat motor neuron diseases: Promises and challenges.

Authors:  Annarita Miccio; Panagiotis Antoniou; Sorana Ciura; Edor Kabashi
Journal:  Mol Ther       Date:  2021-04-03       Impact factor: 11.454
  4 in total

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