Ninth Workshop on Novel Technologies and Gene Transfer for Hemophilia

描述(由申请人提供)： 血友病是一种遗传性凝血障碍，在美国大约有17,000人受到影响。它是由凝血因子VIII或IX的缺陷或缺陷引起的。虽然治疗对许多血友病患者有效，但它包括在出血事件期间或之后使用凝血因子进行终生静脉输注。这种疗法有许多缺点，因此新的疗法已被研究作为治疗血友病的一种手段。血友病的基因治疗将功能凝血因子基因转移到血友病患者的细胞中，使患者的身体能够制造凝血因子蛋白质。在动物实验中，使用各种病毒载体获得凝血因子的持续表达已经取得了相当大的成功。除了以载体为基础的基因转移作为治愈血友病的手段外，对各种新方法的研究仍在继续。许多研究问题仍未得到回答，定期召开的讲习班促进了这一领域的进展，调查人员可以在这些讲习班上讨论其工作的现状。美国国家血友病基金会提议在2007年秋季在一系列基因治疗研讨会中再举办一次。2006年4月的上一次讲习班侧重于血友病的测试和评估；改进动物研究模型；病毒作为基因治疗载体；实验室研究中的基因治疗，包括血友病以外的遗传病的基因治疗；基因治疗的问题；新的凝血因子研究；以及相关临床试验的最新进展。这些领域将在2007年研讨会上作进一步调查。过去的讲习班主要讨论一些非基因转移战略，下一次讲习班的范围将在这方面进一步扩大。主题将包括开发半衰期或活性增加的“设计者”分子，口服给药，基于细胞的治疗，以及可能绕过内在凝血级联中凝血因子VIII或IX缺陷的药物。研讨会为研究人员、临床医生、联邦监管机构、制药公司代表和出血性疾病社区成员之间的公开交流和辩论提供了一个重要机会。研讨会将在费城儿童医院举行。(摘要结束)

DESCRIPTION (provided by applicant): 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)