This application is submitted by the organizers of the 1995 March of Dimes Defects Foundations Clinical Genetics Conference entitled """"""""Novel Approaches to the Treatment of Genetic Disorders."""""""" The meeting is to be held at the Century Plaza Hotel in Los Angeles, California on March 3-4, 1995. Funds are requested to defray the housing and travel costs of 20 graduate students, postdoctoral fellows, and junior investigators so they can attend this conference as active participants. Emphasis during the conference will be given to a wide range of recent treatment approaches for genetic diseases utilized at times ranging from prenatal life to adulthood. General treatment concepts to be described include in utero manipulations, transplantation of stem cells, bone marrow and organs, gene therapy modalities currently in clinical trials, enzyme replacement therapies, and genetic approaches to the treatment of cancer. In addition, recent treatments in practice for specific disorders will be presented, including surgical approaches for skeletal dysplasias (such as leg lengthening in achondroplasia), interventions for management of connective tissue disorders (such as beta blockers for aortic dilatation in Marfan syndrome), and growth hormone therapy for genetic disorders with short stature (such as Down Syndrome). The new area of pharmacologic management of genetic disorders with abnormal behavioral phenotype (such as serotonin-uptake inhibitors for Fragile X syndrome) will be covered. The conference will bring together investigators in a variety of basic and clinical sciences including molecular biology, cellular biology, embryology, developmental biology, cancer biology, clinical genetics, behavioral genetics, birth defects, dysmorphology, oncology, endocrinology and orthopedics. The format includes 20-25 invited speakers, eight platform presentations selected from submitted abstracts, poster sessions, a round table discussion of the ethical aspects of genetic therapies, and a session on contributed cases and diagnostic dilemmas. An audience of 500-600 is anticipated with mechanisms to encourage extensive interaction among the attendees. There will be time reserved for new advances in gene therapy not available at this time. The planners have taken steps to assure the clinical relevance of presentations. Based on the previous experience of these annual meetings over the past 22 years, participation of young investigators in the 1995 conference will contribute significantly to the development of productive collaborations in the years to come. The coorganizers, with help from the other members of the Advisory Committee, will evaluate written applications for travel support and will distribute funds on a competitive basis. The criteria for selecting travel award grantees include submission of an abstract of high quality original research, selection of the research for presentation, and financial need.
