AAV1.NT-3 Gene Therapy for Charcot–Marie–Tooth Neuropathy
نویسندگان
چکیده
منابع مشابه
AAV1.NT-3 Gene Therapy for Charcot–Marie–Tooth Neuropathy
Charcot-Marie-Tooth (CMT) neuropathies represent a heterogeneous group of peripheral nerve disorders affecting 1 in 2,500 persons. One variant, CMT1A, is a primary Schwann cell (SC) disorder, and represents the single most common variant. In previous studies, we showed that neurotrophin-3 (NT-3) improved the trembler(J) (Tr(J)) mouse and also showed efficacy in CMT1A patients. Long-term treatme...
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Fifty percent of patients with long-standing diabetes have numbness, burning, electrical sensations, stabbing, or shooting pain in their feet or legs—and it’s usually worse at night. While the pain is uncomfortable, the lack of sensation can have even worse consequences. If a person’s shoes fit improperly, are too tight, have rough spots inside, or rub when the person walks, the blisters, abras...
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ژورنال
عنوان ژورنال: Molecular Therapy
سال: 2014
ISSN: 1525-0016
DOI: 10.1038/mt.2013.250