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Targeting muscle to treat Charcot-Marie-Tooth disease

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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.
出处 《Neural Regeneration Research》 SCIE CAS CSCD 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。
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