Gene Therapy And Oligonucleotide Compendium

With the potential to prevent, treat or even cure many types of diseases, gene therapy-related research and development continues to be a highly active and growing area within pharmaceutical companies. Currently, there are hundreds of ongoing clinical trials for various gene therapies ranging from inherited genetic disorders to cancer and viral infections and even heart disease and diabetes.

This compendium presents analytical testing methods and workflows for the characterization of gene therapies and genetic vaccines. It includes technical summaries and detailed protocols are presented for purity testing, sizing, sequencing and expression analysis of oligonucleotide-based active agents as well as protein and lipid-based delivery systems.  The aim is to provide scientists with strategies that deliver faster results and more comprehensive information to aid in the research, development and commercialization of gene and cell-based therapeutics and genetic vaccines.