? ? Hemophilia is a genetic disorder of blood coagulation affecting approximately 17,000 individuals in the United States. It is caused by defects or deficiencies in clotting factors VIII or IX. While treatment is effective for many people with hemophilia, it consists of life-long, intravenous infusions with clotting factor administered during or after a bleeding event. This therapy has many drawbacks, and thus novel therapies have been investigated as a means of curing hemophilia. Gene therapy for hemophilia would transfer functioning clotting factor genes into cells in a person with hemophilia, enabling that individual's body to manufacture clotting factor proteins. There has been considerable success in animal studies using various viral vectors to obtain sustained expression of clotting factor. Investigations continue on a variety of novel approaches besides vector-based gene transfer as a means of achieving a cure for hemophilia. Many research questions remain unanswered, and progress in the field is facilitated by regularly convened workshops where investigators can discuss the current state of their work. The National Hemophilia Foundation proposes to hold another in a series of gene therapy workshops in the fall of 2007. The last workshop in April 2006 focused on testing and assessing hemophilia; improving animal research models; viruses as gene therapy delivery vehicles; gene therapy in laboratory studies, including in genetic diseases other than hemophilia; the problems of gene therapy; new clotting factor research; and an update on related clinical trials. These areas will be further investigated at the 2007 workshop. Past workshops featured talks on a number of non-gene transfer strategies, and the scope of the next workshop will be broadened even further in that regard. Topics will include the development of """"""""designer"""""""" molecules with increased half life or activity, oral delivery, cell-based therapies, and drugs that could potentially bypass the factor VIII or IX defects within the intrinsic clotting cascade. The workshop affords an important opportunity for open communication and debate among researchers, clinicians, federal regulators, pharmaceutical company representatives, and members of the bleeding disorders community. The workshop will be held at The Children's Hospital of Philadelphia. (End of Abstract) ? ? ?
