Nature Communications (Mar 2022)

Choice of vector and surgical approach enables efficient cochlear gene transfer in nonhuman primate

  • Eva Andres-Mateos,
  • Lukas D. Landegger,
  • Carmen Unzu,
  • Jean Phillips,
  • Brian M. Lin,
  • Nicholas A. Dewyer,
  • Julio Sanmiguel,
  • Fotini Nicolaou,
  • Michelle D. Valero,
  • Kathrin I. Bourdeu,
  • William F. Sewell,
  • Rudolph J. Beiler,
  • Michael J. McKenna,
  • Konstantina M. Stankovic,
  • Luk H. Vandenberghe

DOI
https://doi.org/10.1038/s41467-022-28969-3
Journal volume & issue
Vol. 13, no. 1
pp. 1 – 10

Abstract

Read online

Gene therapy using Adeno-associated viral vectors (AAV) rescues hearing and balance deficits in mouse models of human disorders. Here, the authors show that AAVAnc80L65 allows efficient cochlear gene transfer in nonhuman primates, and motivate future studies to evaluate gene therapy for hearing and balance disorders.