Thanks to the support of our famiies and friends we were able to fund another worthwhile DBA research project. Dr. David M. Bodine from the NIH is being funded for his research to create and analyze mouse models for some of the mutations that cause Diamond-Blackfan Anemia (DBA). An animal model of DBA will allow an in depth analysis of the defect(s) caused by each of the DBA mutations in untreated animals, allowing the mechanism of the disease to be understood. Once the mechanism has been identified, the effectiveness of conventional therapies can be evaluated and novel therapies designed to target the specific defect can be developed. The mouse model created will be distributed to the DBA community for analysis and the testing and development of therapies for DBA.
Good things come in small packages! I may be little, but I’m a fighter – DBA… no contest.