Question

In blood coagulation, there are many enzymes involved in a cascade of activation reactions to ultimately form fibrin. There is an inherited disorder in Factor X for a small subgroup of the population (bleeding disorder). It is hypothesized that 50% of this different Factor X is defective in the activation site that allows conversion to Factor Xa. You are charged with developing a pharmaceutical (drug) or biopharmaceutical (biologic) to improve this bleeding disorder.

1. Describe what protein or system you will target for drug or biologic design.  

Answer 1

There are two pathways for the process of Coagulation that is the extrinsic and intrinsic pathway. In the extrinsic pathway the factor 7 is converted into factor 7a . In the extrinsic pathway the 7a directly leading to the conversion of 10 factor into 10a which further convert the 11 to 11a and then the fibrinogen is converted into fibrin .In the intrinsic pathway the 12 factor is converted to 12a ,then the 11 is converted into 11a ,then 9 is converted to 9a and the 10 is converted to 10a and then the cycle continues as that of extrinsic pathway cycle. In a defective factor X ,the active site of this factor is defective that is factor 9a and then 7a through the intrinsic and extrinsic pathway cannot be able to convert the factor X into its active form. Therefore ,the 9a and 7a pathway is targeted by the drug as explained which is the pathway for the conversion of factor X into its active form that is important for coagulation.

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