Platform for Delivering Targeted Therapies to Treat Neurological Diseases

The progressive loss of function of neurons is the basis for most neurodegener-ative diseases, including AlzheimerΓÇÖs disease, ParkinsonΓÇÖs disease, amyotrophic lateral sclerosis and HuntingtonΓÇÖs disease, the transmissible spongiform encephalopathies (TSEs or prion diseases), and botulism, caused by bacterial botulinum neurotoxins that bind to and enter neurons. There is a need for therapeutics that slow or prevent the progression of these diseases. Technology 1706 is a platform that can be used to deliver therapeutic drugs/molecules into neurons. The foundations of this platform are engineered AB5 toxins obtained from bacteria. AB5 toxins possess two major components: B and A. The binding domain (B) recognizes and binds to specific gangliosides on the cell surface. This binding facilitates the endocytosis of catalytic domain (A), an enzyme which is respon-sible for the toxic effects. Joseph Hill jhill@mcw.edu 414-955-4381

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