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Gene therapy for the treatment of X-linked retinitis pigmentosa.
Martinez-Fernandez De La Camara C, Nanda A, Salvetti AP, Fischer MD, MacLaren RE. Martinez-Fernandez De La Camara C, et al. Expert Opin Orphan Drugs. 2018 Feb 27;6(3):167-177. doi: 10.1080/21678707.2018.1444476. Expert Opin Orphan Drugs. 2018. PMID: 30057863 Free PMC article.
Molecular Strategies for RPGR Gene Therapy.
Cehajic Kapetanovic J, McClements ME, Martinez-Fernandez de la Camara C, MacLaren RE. Cehajic Kapetanovic J, et al. Genes (Basel). 2019 Sep 4;10(9):674. doi: 10.3390/genes10090674. Genes (Basel). 2019. PMID: 31487940 Free PMC article. Review.
RPGR gene therapy presents challenges in cloning the coding sequence.
Martinez-Fernandez De La Camara C, Cehajic-Kapetanovic J, MacLaren RE. Martinez-Fernandez De La Camara C, et al. Expert Opin Biol Ther. 2020 Jan;20(1):63-71. doi: 10.1080/14712598.2020.1680635. Epub 2019 Oct 20. Expert Opin Biol Ther. 2020. PMID: 31612744 Free PMC article. Review.
Initial results from a first-in-human gene therapy trial on X-linked retinitis pigmentosa caused by mutations in RPGR.
Cehajic-Kapetanovic J, Xue K, Martinez-Fernandez de la Camara C, Nanda A, Davies A, Wood LJ, Salvetti AP, Fischer MD, Aylward JW, Barnard AR, Jolly JK, Luo E, Lujan BJ, Ong T, Girach A, Black GCM, Gregori NZ, Davis JL, Rosa PR, Lotery AJ, Lam BL, Stanga PE, MacLaren RE. Cehajic-Kapetanovic J, et al. Nat Med. 2020 Mar;26(3):354-359. doi: 10.1038/s41591-020-0763-1. Epub 2020 Feb 24. Nat Med. 2020. PMID: 32094925 Free PMC article. Clinical Trial.
25 results