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Carey, Megan

Major: 
Biology
Mentor: 
Dr. Pudur Jagadeeswaran
Current Research Topic: 
CRISPR/Cas9 Knockout of Factor IX in Danio rerio to Model Hemophilia B
Abstract: 

Current therapies for hemophilia B involve recombinant factor IX or the introduction of various forms of exogenous concentrates of factor IX to afflicted patients. These treatments become ineffective, however, if the patient forms an immune response and rejects factor IX. Production of antibodies called Inhibitors, or anti-FIX, by the immune system occurs in some severe cases of hemophilia B. At present there are only limited studies to correct the immune response such as the attempts by neonatal gene therapy in mice. Due to the uncertainty of success by current treatments, another model organism is needed to study and develop alternate therapies. This study uses the CRISPR/Cas9 nuclease system to knockout factor IX in zebrafish embryos. In doing so, we predict that knockout of the f9a and f9b genes will result in hemophilic zebrafish that can be used as an additional model organism for hemophilia B and aid in the development of future therapies.