Targeting muscle to treat Charcot-Marie-Tooth disease
作者机构:Department of Neuromuscular Diseases and UCL Queen Square Motor Neuron Disease CentreUCL Queen Square Institute of NeurologyUniversity College LondonLondonUK UK Dementia Research InstituteUniversity College LondonLondonUK
出 版 物:《Neural Regeneration Research》 (中国神经再生研究(英文版))
年 卷 期:2024年第19卷第8期
页 面:1653-1654页
核心收录:
学科分类:0710[理学-生物学] 1002[医学-临床医学] 1001[医学-基础医学(可授医学、理学学位)] 100204[医学-神经病学] 10[医学]
基 金:supported by the funding from the Medical Research Council(MR/S006990/1) the Wellcome Trust(103191/A/13/Z) the Rosetrees Trust(M806) the UCL Neurogenetic Therapies Programme funded by The Sigrid Rausing Trust named inventor on patent GB2303495.2(patent applicant,UCL Business Ltd.,status pending) which describes and protects AAV-BDNF technology for treatment of CMT
主 题:Marie sensation neuropathy
摘 要:Charcot-Marie-Tooth disease(CMT)is a hereditary peripheral neuropathy causing muscle weakness/wasting and sensory dysfunction predominantly in limb *** patients display gait abnormalities,foot deformities,loss of sensation and decreased/absent deep tendon reflexes,with motor symptoms usually being more prominent than sensory.