The field of gene therapy continues to evolve as advances are made in vector development, vector-host cell interactions and understanding of disease pathophysiology. As barriers to successful human gene therapy continue to be addressed at the molecular and cellular level, the research field is progressing towards clinical applications which are beginning to define both the safety of this therapeutic approach and its impact on a variety of diseases. These clinical models are helping investigators define new challenges, including vector production and quality control, in vivo assays to detect gene expression, safety measurements and clinical endpoints. The goal of this conference is to highlight progress in molecular and cellular biology and to establish a basic framework for clinical trial development in human gene therapy. The results of recent clinical trials will be presented. Major barriers to human studies, including limitations of current vectors, host immune response, stem cell biology and disease pathogenesis will be discussed. The meeting will bring together basic scientists and clinical investigators to face these challenges. The program will focus on innovative technologies in vector development, vector-host interactions, challenges of clinical trial development, genetic diseases, stem cell biology, cardiovascular diseases, CNS disorders, AIDS, cancer and vaccine development. Each session will highlight the basic science, preclinical studies and review current clinical trials in progress. The program of speakers emphasizes representation of women and young investigators. The mix of speakers is designed to expose predoctoral and postdoctoral attendees to prominent exponents of each of the major gene therapy methodologies, providing developing scientists the tools for making informed choices in defining their research directions. A portion of the funding sought will be used to defray costs incurred by graduate students and postdoctoral fellows who apply to attend the meeting. Selection of award recipients will be made based on the quality of abstracts submitted for the poster presentation.
