作者: Tanaz Sharifnia , Andrew L. Hong , Corrie A. Painter , Jesse S. Boehm
DOI: 10.1016/J.CHEMBIOL.2017.08.002
关键词: Biology 、 Clinical settings 、 Patient accrual 、 CRISPR 、 Rare cancer 、 Profiling (information science) 、 Bioinformatics 、 Computational biology 、 Genomics 、 Basic research 、 Cas9
摘要: Rare cancers pose unique challenges to research due their low incidence. Barriers include a scarcity of tissue and experimental models enable basic insufficient patient accrual for clinical studies. Consequently, an understanding the genetic cellular features many rare cancer types associated vulnerabilities has been lacking. However, new opportunities are emerging facilitate discovery therapeutic targets in cancers. Online platforms allowing patients with organize on unprecedented scale, tumor genome sequencing is now routinely performed settings, efficiency patient-derived model generation improved. New CRISPR/Cas9 small-molecule libraries permit dependency rapid systematic fashion. In parallel, large-scale studies common provide reference datasets help interpret profiling data. Together, these advances motivate consideration frameworks accelerate target discovery.