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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.
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.
Successful subretinal delivery and monitoring of MicroBeads in mice.
Fischer MD, Goldmann T, Wallrapp C, Mühlfriedel R, Beck SC, Stern-Schneider G, Ueffing M, Wolfrum U, Seeliger MW. Fischer MD, et al. PLoS One. 2013;8(1):e55173. doi: 10.1371/journal.pone.0055173. Epub 2013 Jan 28. PLoS One. 2013. PMID: 23383096 Free PMC article.