Targeting muscle to treat Charcot-Marie-Tooth disease  

作　　者：David Villarroel-Campos James N.Sleigh 

机构地区：[1]Department of Neuromuscular Diseases and UCL Queen Square Motor Neuron Disease Centre,UCL Queen Square Institute of Neurology,University College London,London,UK [2]UK Dementia Research Institute,University College London,London,UK

出　　处：《Neural Regeneration Research》2024年第8期1653-1654,共2页中国神经再生研究(英文版)

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。

摘　　要：Charcot-Marie-Tooth disease(CMT)is a hereditary peripheral neuropathy causing muscle weakness/wasting and sensory dysfunction predominantly in limb extremities.CMT patients display gait abnormalities,foot deformities,loss of sensation and decreased/absent deep tendon reflexes,with motor symptoms usually being more prominent than sensory.

关 键 词：Marie SENSATION NEUROPATHY 

分 类 号：R746.4[医药卫生—神经病学与精神病学]