Molecular Pathways: Understanding and Targeting Mutant Spliceosomal Proteins.

ABSTRACT: Splicing of precursor messenger RNA is a critical step in regulating gene expression, and major advances are being made in understanding the composition and structure of the enzymatic complex that performs splicing, which is termed the "spliceosome." In parallel, there has been increased appreciation for diverse mechanisms by which alterations in splicing contribute to cancer pathogenesis. Key among these include change-of-function mutations in genes encoding spliceosomal proteins. Such mutations are among the most common genetic alterations in myeloid and lymphoid leukemias, making efforts to therapeutically target cells bearing these mutations critical. To this end, recent studies have clarified that pharmacologic modulation of splicing may be preferentially lethal for cells bearing spli

SUBMITTER: Yoshimi A 

PROVIDER: S-EPMC5241248 | biostudies-literature | 2017 Jan

REPOSITORIES: biostudies-literature