Gene therapy for RPE65-mediated retinal dystrophies
- PMID: 29248533
- DOI: 10.1016/j.survophthal.2017.12.006
Gene therapy for RPE65-mediated retinal dystrophies
Comment on
-
Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial.Lancet. 2017 Aug 26;390(10097):849-860. doi: 10.1016/S0140-6736(17)31868-8. Epub 2017 Jul 14. Lancet. 2017. PMID: 28712537 Free PMC article. Clinical Trial.
Similar articles
-
[RPE65-associated retinal dystrophies: gene therapy preserves vision].MMW Fortschr Med. 2022 Feb;164(Suppl 4):40-41. doi: 10.1007/s15006-022-0765-7. MMW Fortschr Med. 2022. PMID: 35146717 Review. German. No abstract available.
-
CLINICAL PROGRESS IN INHERITED RETINAL DEGENERATIONS: GENE THERAPY CLINICAL TRIALS AND ADVANCES IN GENETIC SEQUENCING.Retina. 2017 Mar;37(3):417-423. doi: 10.1097/IAE.0000000000001341. Retina. 2017. PMID: 27753762 Free PMC article. Review.
-
Rpe65 as a modifier gene for inherited retinal degeneration.Eur J Neurosci. 2006 Feb;23(4):1028-34. doi: 10.1111/j.1460-9568.2006.04639.x. Eur J Neurosci. 2006. PMID: 16519667 Free PMC article.
-
RPE65 gene delivery restores isomerohydrolase activity and prevents early cone loss in Rpe65-/- mice.Invest Ophthalmol Vis Sci. 2006 Mar;47(3):1177-84. doi: 10.1167/iovs.05-0965. Invest Ophthalmol Vis Sci. 2006. PMID: 16505056
-
Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness.Mol Ther. 2005 Dec;12(6):1072-82. doi: 10.1016/j.ymthe.2005.08.008. Epub 2005 Oct 14. Mol Ther. 2005. PMID: 16226919 Free PMC article.
Cited by
-
The Role of Retinal Pigment Epithelial Cells in Age-Related Macular Degeneration: Phagocytosis and Autophagy.Biomolecules. 2023 May 29;13(6):901. doi: 10.3390/biom13060901. Biomolecules. 2023. PMID: 37371481 Free PMC article. Review.
Publication types
MeSH terms
Substances
LinkOut - more resources
Full Text Sources
Other Literature Sources