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Letter to the Editor

Letter to the editor: ‘Emerging gene therapy products for RPGR-associated X-linked retinitis pigmentosa’

, , ORCID Icon, &
Pages 445-447 | Received 05 Nov 2019, Accepted 17 Dec 2019, Published online: 23 Dec 2022

References

  • Martinez-Fernandez de la Camara C, Cehajic Kapetanovic J, MacLaren RE. Emerging gene therapy products for RPGR-associated X-linked retinitis pigmentosa. Expert Opin Emerg Drugs. 2022;27(4):429–441. DOI:10.1080/14728214.2022.2152003
  • Karra D, Jacobi FK, Broghammer M, et al. Population haplotypes of exon ORF15 of the retinitis pigmentosa GTPase regulator gene in Germany. Mol Diag Ther. 2006;10(2):115–123.
  • Demirci FYK, Rigatti BW, Wen G, et al. X-Linked Cone-Rod Dystrophy (Locus COD1): identification of mutations in RPGR exon ORF15. Am J Hum Genet. 2022;70:1049–1053.
  • Sun X, Park JH, Gumerson J, et al. Loss of RPGR glutamylation underlies the pathogenic mechanism of retinal dystrophy caused by TTLL5 mutations. Proc Natl Acad Sci U S A. 2016;113(21):E2925–34.
  • Pawlyk BS, Bulgakov OV, Sun X, et al. Photoreceptor rescue by an abbreviated human RPGR gene in a murine model of X-linked retinitis pigmentosa. Gene Ther. 2015;23(2):196–204.
  • West EL, Majumder P, Naeem A, et al. Antioxidant and lipid supplementation improve the development of photoreceptor outer segments in pluripotent stem cell-derived retinal organoids. Stem Cell Reports. 2022;17(4):775–788.
  • Hong DH, Pawlyk BS, Adamian M, et al. Dominant, gain-of-function mutant produced by truncation of RPGR. Invest Ophthalmol Vis Sci. 2004;45(1):36–41.
  • Hong DH, Pawlyk BS, Adamian, M, et al. A single, abbreviated RPGR-ORF15 variant reconstitutes RPGR function in vivo. Invest Ophthalmol Vis Sci. 2005;46(2):435–441.

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