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      RNA and disease.

      1 , ,
      Cell
      Elsevier BV

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          Abstract

          Cellular functions depend on numerous protein-coding and noncoding RNAs and the RNA-binding proteins associated with them, which form ribonucleoprotein complexes (RNPs). Mutations that disrupt either the RNA or protein components of RNPs or the factors required for their assembly can be deleterious. Alternative splicing provides cells with an exquisite capacity to fine-tune their transcriptome and proteome in response to cues. Splicing depends on a complex code, numerous RNA-binding proteins, and an enormously intricate network of interactions among them, increasing the opportunity for exposure to mutations and misregulation that cause disease. The discovery of disease-causing mutations in RNAs is yielding a wealth of new therapeutic targets, and the growing understanding of RNA biology and chemistry is providing new RNA-based tools for developing therapeutics.

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          Author and article information

          Journal
          Cell
          Cell
          Elsevier BV
          1097-4172
          0092-8674
          Feb 20 2009
          : 136
          : 4
          Affiliations
          [1 ] Department of Pathology, Baylor College of Medicine, Houston, TX 77030, USA. tcooper@bcm.tmc.edu
          Article
          S0092-8674(09)00148-2 HHMIMS194224
          10.1016/j.cell.2009.02.011
          2866189
          19239895
          2bbc3639-855f-47e1-ae11-be05c7282bd8
          History

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