Subretinal gene therapy delays vision loss in a Bardet-Biedl Syndrome type 10 mouse model.
Hsu Y, Bhattarai S, Thompson JM, Mahoney A, Thomas J, Mayer SK, Datta P, Garrison J, Searby CC, Vandenberghe LH, Seo S, Sheffield VC, Drack AV.
Hsu Y, et al. Among authors: thompson jm.
Mol Ther Nucleic Acids. 2022 Dec 12;31:164-181. doi: 10.1016/j.omtn.2022.12.007. eCollection 2023 Mar 14.
Mol Ther Nucleic Acids. 2022.
PMID: 36700052
Free PMC article.
In this study, we performed gene augmentation therapy by injecting a viral construct subretinally to deliver the coding sequence of the mouse Bbs10 gene to treat retinal degeneration in a BBS10 mouse model. Long-term efficacy was assessed by measuring the electrical functi …
In this study, we performed gene augmentation therapy by injecting a viral construct subretinally to deliver the coding sequence of the mous …