Hematologic Diseases in the Post-Genome Era: New Insights, New Therapies, New Possibilities
The past decade of scientific investigation has led to new understanding of somatic genome mutations and their role in hematologic diseases, as well as important insights into the role of germline mutations and epigenetic drivers of cancers such as leukemia. In each of these areas, the relative pristine genomes of pediatric cancers and the successes in treating monogenic diseases in children with genetically modified cells and targeting the epigenome have yielded insights and tools that have and will be used in adult diseases. During the 2015 ASH Presidential Symposium, three experts will discuss the insights gained in the study of childhood diseases and their impact on evolving therapies for adult hematologic disorders.
Dr. Mignon Loh will describe how germline and somatic mutations in juvenile myelomonocytic leukemia have led to new understanding of the evolution of this disease and have provided insights into the complexity of targeting RAS-mutated leukemias.
Dr. Scott Armstrong will discuss how the understanding of epigenetic lesions in infant and childhood acute leukemias is leading to new therapeutic approaches in both pediatric and adult leukemia.
Dr. Luigi Naldini will discuss how nearly 20 years of research in gene therapy has set the stage for gene replacement and editing in pediatric monogenic diseases, such as primary immunodeficiencies and storage diseases, and will lead the way to new therapies for adult hematologic diseases.