preclinical Testing of Novel Therapies in FA

$278,816P01FY2014HLNIH

Oregon Health & Science University, Portland OR

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Abstract

FA is a rare disease and about 500 patients with FA are alive in the U.S.A. The 3 main clinical features of FA are birth defects (missing thumb/radius; microphthalmia; cafe-au-lait spots etc.), progressive marrow failure and cancer suscepfibility [1]. In FA. the life span Is severely shortened [2]. The main cause of death is bone marrow failure with the associated complications of anemia, bleeding and infecfion. Another cause of death is cancer, particulariy acute myelogenous leukemia, but also solid tumors, particulariy squamous cell carcinomas [3].

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