Expanding the Atlas of Functional Missense Variation for Human Genes

نویسندگان

  • Jochen Weile
  • Song Sun
  • Atina G. Cote
  • Jennifer Knapp
  • Marta Verby
  • Joseph Mellor
  • Yingzhou Wu
  • Carles Pons
  • Cassandra Wong
  • Natascha van Lieshout
  • Fan Yang
  • Murat Tasan
  • Guihong Tan
  • Shan Yang
  • Douglas M Fowler
  • Robert Nussbaum
  • Jesse D. Bloom
  • Marc Vidal
  • David E Hill
  • Patrick Aloy
  • Frederick P. Roth
چکیده

Although we now routinely sequence human genomes, we can confidently identify only a fraction of the sequence variants that have a functional impact. Here we developed a deep mutational scanning framework that produces exhaustive maps for human missense variants by combining random codon-mutagenesis and multiplexed functional variation assays with computational imputation and refinement. We applied this framework to four proteins corresponding to six human genes: UBE2I (encoding SUMO E2 conjugase), SUMO1 (small ubiquitin-like modifier), TPK1 (thiamin pyrophosphokinase), and CALM1/2/3 (three genes encoding the protein calmodulin). The resulting maps recapitulate known protein features, and confidently identify pathogenic variation. Assays potentially amenable to deep mutational scanning are already available for 57% of human disease genes, suggesting that DMS could ultimately map functional variation for all human disease genes.

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تاریخ انتشار 2017