Glycoviruses: Chemical Glycosylation Retargets Adenoviral Gene Transfer

摘要

Gene therapy of infectious, vascular, and multifactorial diseases employs a variety of viruses, which each have specific qualities that make them suitable for their chosen application. Gene therapy provides a means to exploit knowledge generated under the human genome project by the use of gene delivery vectors to supplement the function of missing or mutated genes. Some applications of gene therapy require therapeutic gene delivery to specific diseased cells, such as the cystic fibrotic epithelia for treatment of cystic fibrosis, whereas others accommodate transgene expression within nondiseased cells such as muscle cells or liver hepatocytes in a so-called cell factory approach. In both applications, successful delivery of the virus requires precise target-cell specificity, an ability to evade neutralizing antibodies, and increased blood circulation to the target cell or tissue. Control of these properties is one of the major challenges facing viral gene therapy today.