Cornell Research Foundation Inc.

The Feinstein Institutes for Medical Research

The Feinstein for Medical Research

Philippe Marambaud

Fabien Campagne

Methods and pharmaceutical compositions comprising one or more mTOR inhibitors such as sirolimus and one or more receptor tyrosine kinase inhibitors such as nintedanib are disclosed for treating vascular lesions and hereditary hemorrhagic telangiectasia.

Combined sirolimus and nintedanib therapy for vascular lesions and hereditary hemorrhagic telangiectasia

Provided are methods of screening test compounds for the ability to alter calcium homeostasis in mammalian cells by experimentally determining if a compound affects CALHM1, CALHM2, or CALHM3 expression or activity. Additionally provided are methods of screening a test compound for the ability to inhibit ERK 1/2 phosphorylation in a mammalian cell. Further provided are methods of screening a test compound for the ability to inhibit amyloid-beta peptide accumulation in a mammalian cell or biological fluid, methods of screening for a test compound that may affect Alzheimer's disease and methods of determining the likelihood that a subject will be diagnosed with Alzheimer's disease. Also provided are isolated and purified mammalian CALHM proteins, vectors comprising a nucleic acid sequence encoding the CALHM1, CALHM2, and CALHM3 proteins, and mammalian cells transfected with the vectors. Additionally, methods of affecting Ca2+ levels in a mammalian cell are provided.

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Astoria, NY, United States of America

Philippe Marambaud