Nature Communications (Oct 2022)

Cell-specific regulation of gene expression using splicing-dependent frameshifting

  • Jonathan P. Ling,
  • Alexei M. Bygrave,
  • Clayton P. Santiago,
  • Rogger P. Carmen-Orozco,
  • Vickie T. Trinh,
  • Minzhong Yu,
  • Yini Li,
  • Ying Liu,
  • Kyra D. Bowden,
  • Leighton H. Duncan,
  • Jeong Han,
  • Kamil Taneja,
  • Rochinelle Dongmo,
  • Travis A. Babola,
  • Patrick Parker,
  • Lizhi Jiang,
  • Patrick J. Leavey,
  • Jennifer J. Smith,
  • Rachel Vistein,
  • Megan Y. Gimmen,
  • Benjamin Dubner,
  • Eric Helmenstine,
  • Patric Teodorescu,
  • Theodoros Karantanos,
  • Gabriel Ghiaur,
  • Patrick O. Kanold,
  • Dwight Bergles,
  • Ben Langmead,
  • Shuying Sun,
  • Kristina J. Nielsen,
  • Neal Peachey,
  • Mandeep S. Singh,
  • W. Brian Dalton,
  • Fatemeh Rajaii,
  • Richard L. Huganir,
  • Seth Blackshaw

DOI
https://doi.org/10.1038/s41467-022-33523-2
Journal volume & issue
Vol. 13, no. 1
pp. 1 – 14

Abstract

Read online

Precise and reliable gene delivery remains technically challenging. Here, the authors show that rationally designed frameshifting splicing can be used to express genes only in targeted cell types, with the potential to enhance the specificity AAV gene delivery.