New genome-wide functional analysis tools needed to accelerate drug development

摘要

Since the first human genome was sequenced over 10 years ago, a tremendous amount of data has been collected and associated with biological traits, responses, and disease processes. While this information has provided a basis for many new diagnostics, it has not stimulated significant development of novel therapeutics. For new drug development, a more sophisticated understanding of how genes function is required. While there are some techniques that can be used to investigate the function of individual genes, few options exist for genome-wide functional analysis. Only two technologies currently form the basis of techniques for broad-based genetic screens that assess the functions of large numbers of genes in a single assay: RNA interference and CRISPR knockout screens. To make real progress in understanding the disease process and identifying novel therapeutic approaches, new more powerful methods are required.