strategy for treating motor neuron diseases using a fusion protein of botulinum toxin binding domain and streptavidin for viral vector access work in progress治疗运动神经元疾病的策略使用肉毒杆菌毒素的融合蛋白绑定域和链霉亲和素对病毒载体访问工作进展.pdfVIP

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strategy for treating motor neuron diseases using a fusion protein of botulinum toxin binding domain and streptavidin for viral vector access work in progress治疗运动神经元疾病的策略使用肉毒杆菌毒素的融合蛋白绑定域和链霉亲和素对病毒载体访问工作进展.pdf

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strategy for treating motor neuron diseases using a fusion protein of botulinum toxin binding domain and streptavidin for viral vector access work in progress治疗运动神经元疾病的策略使用肉毒杆菌毒素的融合蛋白绑定域和链霉亲和素对病毒载体访问工作进展

Toxins 2010, 2, 2872-2889; doi:10.3390/toxins2122872 OPEN ACCESS toxins ISSN 2072-6651 /journal/toxins Article Strategy for Treating Motor Neuron Diseases Using a Fusion Protein of Botulinum Toxin Binding Domain and Streptavidin for Viral Vector Access: Work in Progress Daniel B. Drachman 1,*, Robert N. Adams 1, Uma Balasubramanian 1 and Yang Lu 2 1 Department of Neurology, Johns Hopkins School of Medicine, 600 North Wolfe Ave, Baltimore, MD 21287, USA; E-Mails: rna@ (R.N.A.); uma1@ (U.B.) 2 Sirnaomics, Inc., 401 Professional Dr. Suite 130, Gaithersburg, MD 20879, USA; E-Mail: alanlu@ * Author to whom correspondence should be addressed; E-Mail: dandrac@; Tel.: +1-410-955-5406; Fax: +1-410-955-1961. Received: 11 October 2010; in revised form: 16 December 2010 / Accepted: 17 December 2010 / Published: 20 December 2010 Abstract: Although advances in understanding of the pathogenesis of amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy (SMA) have suggested attractive treatment strategies, delivery of agents to motor neurons embedded within the spinal cord is problematic. We have designed a strategy based on the specificity of botulinum toxin, to direct entry of viral vectors carrying candidate therapeutic genes into motor neurons. We have engineered and expressed fusion proteins consisting of the binding domain of botulinum toxin type A fused to streptavidin (SAv). This fusion protein will direct biotinylated viral vectors carrying therapeu