Targeting muscle to treat Charcot-Marie-Tooth disease

作     者：David Villarroel-Campos James N.Sleigh David Villarroel-Campos;James N.Sleigh

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.