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Cell Chem Biol DOI:10.1016/j.chembiol.2017.08.002

Emerging Opportunities for Target Discovery in Rare Cancers.

Publication TypeJournal Article
Year of Publication2017
AuthorsSharifnia, T, Hong, AL, Painter, CA, Boehm, JS
JournalCell Chem Biol
Date Published2017 Sep 21

Rare cancers pose unique challenges to research due to their low incidence. Barriers include a scarcity of tissue and experimental models to enable basic research and insufficient patient accrual for clinical studies. Consequently, an understanding of the genetic and cellular features of many rare cancer types and their associated vulnerabilities has been lacking. However, new opportunities are emerging to facilitate discovery of therapeutic targets in rare cancers. Online platforms are allowing patients with rare cancers to organize on an unprecedented scale, tumor genome sequencing is now routinely performed in research and clinical settings, and the efficiency of patient-derived model generation has improved. New CRISPR/Cas9 and small-molecule libraries permit cancer dependency discovery in a rapid and systematic fashion. In parallel, large-scale studies of common cancers now provide reference datasets to help interpret rare cancer profiling data. Together, these advances motivate consideration of new research frameworks to accelerate rare cancer target discovery.


Alternate JournalCell Chem Biol
PubMed ID28938087