Publisher's Synopsis
Enormous technical advances in the field of eukaryotic genetics have provided the opportunity to study human biology as intensively as previous studies of prokaryotic biology. However, despite spectacular progress in our understanding of the molecular basis of human genetic diseases, they usually remain difficult to cure, and often fatal in the pediatric age group. This volume is timely in addressing new genetic approaches to studying and treating intractable diseases with known and unknown etiology.;A first step in many experiments is the isolation of genes responsible for disease. The function and regulation of these genes can then be studied in vivo using the powerful techniques of mouse genetics described here so eloquently. Mouse models are invaluable in the study of immunological disorders, neuropathy, enzyme deficiency and metabolic diseases. Technology used for developing animal models of human diseases has tremendous potential for the definition of gene function and the study of developmental processes. This volume addresses possible clinical applications of gene manipulation at present and in the future. Ultimately these studies aim to increase understanding of the disease process and lead to more rational approaches to drug therapy, and secondly to refine therapies based on somatic gene transfer.
