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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.
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.
Molecular Therapies for Choroideremia.
Cehajic Kapetanovic J, Barnard AR, MacLaren RE. Cehajic Kapetanovic J, et al. Genes (Basel). 2019 Sep 23;10(10):738. doi: 10.3390/genes10100738. Genes (Basel). 2019. PMID: 31548516 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.
Progress in the development of novel therapies for choroideremia.
Cehajic Kapetanovic J, Patrício MI, MacLaren RE. Cehajic Kapetanovic J, et al. Expert Rev Ophthalmol. 2019;14(6):277-285. doi: 10.1080/17469899.2019.1699406. Epub 2019 Dec 26. Expert Rev Ophthalmol. 2019. PMID: 32002021 Free PMC article.
48 results