Comprehensive identification of mRNA isoforms reveals the diversity of neural cell-surface molecules with roles in retinal development and disease

Nat Commun. 2020 Jul 3;11(1):3328. doi: 10.1038/s41467-020-17009-7.

Abstract

Genes encoding cell-surface proteins control nervous system development and are implicated in neurological disorders. These genes produce alternative mRNA isoforms which remain poorly characterized, impeding understanding of how disease-associated mutations cause pathology. Here we introduce a strategy to define complete portfolios of full-length isoforms encoded by individual genes. Applying this approach to neural cell-surface molecules, we identify thousands of unannotated isoforms expressed in retina and brain. By mass spectrometry we confirm expression of newly-discovered proteins on the cell surface in vivo. Remarkably, we discover that the major isoform of a retinal degeneration gene, CRB1, was previously overlooked. This CRB1 isoform is the only one expressed by photoreceptors, the affected cells in CRB1 disease. Using mouse mutants, we identify a function for this isoform at photoreceptor-glial junctions and demonstrate that loss of this isoform accelerates photoreceptor death. Therefore, our isoform identification strategy enables discovery of new gene functions relevant to disease.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Amino Acid Sequence
  • Animals
  • Base Sequence
  • Gene Expression Profiling / methods
  • Gene Expression Regulation, Developmental
  • Genetic Variation*
  • Humans
  • Membrane Proteins / genetics*
  • Membrane Proteins / metabolism
  • Mice, Inbred C57BL
  • Mice, Inbred Strains
  • Mice, Knockout
  • Nerve Tissue Proteins / genetics
  • Nerve Tissue Proteins / metabolism
  • Photoreceptor Cells, Vertebrate / metabolism*
  • RNA Isoforms / genetics*
  • RNA Isoforms / metabolism
  • Retina / cytology
  • Retina / growth & development
  • Retina / metabolism*
  • Retinal Degeneration / genetics*
  • Retinal Degeneration / metabolism
  • Sequence Homology, Amino Acid
  • Sequence Homology, Nucleic Acid

Substances

  • Crb1 protein, mouse
  • Membrane Proteins
  • Nerve Tissue Proteins
  • RNA Isoforms