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.
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。