Spinal muscular atrophy: From approved therapies to future therapeutic targets for personalized medicine
Author:
Publisher
Elsevier BV
Subject
General Biochemistry, Genetics and Molecular Biology
Reference154 articles.
1. Prevalence, incidence and carrier frequency of 5q-linked spinal muscular atrophy - a literature review;Verhaart;Orphanet J. Rare Dis.,2017
2. Identification and characterization of a spinal muscular atrophy-determining gene;Lefebvre;Cell,1995
3. A single nucleotide difference that alters splicing patterns distinguishes the SMA gene SMN1 from the copy gene SMN2;Monani;Hum. Mol. Genet.,1999
4. An exonic enhancer is required for inclusion of an essential exon in the SMA-determining gene SMN;Lorson;Hum. Mol. Genet.,2000
5. The role of survival motor neuron protein (SMN) in protein homeostasis;Chaytow;Cell. Mol. Life Sci.,2018
Cited by 78 articles. 订阅此论文施引文献 订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献
1. Subacute liver injury in two young infants following gene replacement therapy for spinal muscular atrophy;Molecular Therapy - Methods & Clinical Development;2024-09
2. Transforming care for spinal muscular atrophy: A critical look at treatment paradigms;Molecular Therapy;2024-08
3. Changes in abilities over the initial 12 months of nusinersen treatment for type II SMA;Neuromuscular Disorders;2024-08
4. Recent Progress in Gene-Targeting Therapies for Spinal Muscular Atrophy: Promises and Challenges;Genes;2024-07-30
5. Gene therapy for spinal muscular atrophy: perspectives on the possibility of optimizing SMN1 delivery to correct all neurological and systemic perturbations;Neural Regeneration Research;2024-07-29
1.学者识别学者识别
2.学术分析学术分析
3.人才评估人才评估
"同舟云学术"是以全球学者为主线，采集、加工和组织学术论文而形成的新型学术文献查询和分析系统，可以对全球学者进行文献检索和人才价值评估。用户可以通过关注某些学科领域的顶尖人物而持续追踪该领域的学科进展和研究前沿。经过近期的数据扩容，当前同舟云学术共收录了国内外主流学术期刊6万余种，收集的期刊论文及会议论文总量共计约1.5亿篇，并以每天添加12000余篇中外论文的速度递增。我们也可以为用户提供个性化、定制化的学者数据。欢迎来电咨询！咨询电话：010-8811{复制后删除}0370
www.globalauthorid.com
TOP
Copyright © 2019-2024 北京同舟云网络信息技术有限公司 京公网安备11010802033243号 京ICP备18003416号-3