Gene delivery to cone photoreceptors by subretinal injection of rAAV2/6 in the mouse retina

Biochem Biophys Res Commun. 2019 Jul 12;515(1):222-227. doi: 10.1016/j.bbrc.2019.05.117. Epub 2019 May 27.


Adeno-associated virus (AAV) has been studied as a safe delivery tool for gene therapy of retinal blinding diseases such as Leber's congenital amaurosis (LCA). The tropism of recombinant AAV (rAAV) including its specificity and efficiency in targeting retinal cell types has been studied with native or engineered capsids, along with specific promoters. However, one of the rAAV serotypes, rAAV2/6, has not been well-studied based on a report of low infection efficiency in the retina. We investigated the tropism of several rAAVs by subretinal injection in the adult mouse and found that rAAV2/6 predominantly infected cone photoreceptors including the main spectral type. Our data suggest that subretinal injection with rAAV2/6 may provide both an efficacious and specific means of gene delivery to cone photoreceptors in murine retinas.

Keywords: Adeno-associated virus; Cone photoreceptors; Gene therapy; The mammalian retina.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Dependovirus / genetics*
  • Genetic Therapy / methods*
  • Genetic Vectors / administration & dosage
  • Genetic Vectors / genetics*
  • Injections
  • Leber Congenital Amaurosis / genetics
  • Leber Congenital Amaurosis / therapy
  • Mice, 129 Strain
  • Opsins / genetics
  • Opsins / metabolism
  • Retina / metabolism*
  • Retina / virology
  • Retinal Cone Photoreceptor Cells / metabolism*
  • Retinal Cone Photoreceptor Cells / virology
  • Retinal Diseases / genetics
  • Retinal Diseases / therapy*
  • Treatment Outcome


  • Opsins