Gene therapy success depends on ability to advance viral delivery vectors to commercialization
Wednesday, May 18, 2011 - 12:30
in Biology & Nature
Many gene therapy strategies designed to deliver a normal copy of a gene to cells carrying a disease-causing genetic mutation rely on a modified virus to transfer the gene product into affected tissues. One technology platform that is well suited for in vivo delivery of genes is based on adeno-associated viruses (AAV). As these novel therapies move closer to commercialization, so do the methods for large-scale production and efficient delivery of AAV vectors, which are documented in a series of articles published online ahead of print in Human Gene Therapy, a peer-reviewed journal published by Mary Ann Liebert, Inc.