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Year Number of Results
2013 3
2014 1
2015 1
2016 1
2017 1
2018 2
2019 5
2020 3
2021 3
2022 3
2023 3
2024 1

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Page 1
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. Among authors: martinez fernandez de la camara c. 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. Among authors: martinez fernandez de la camara c. 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.
Outcomes and Adverse Effects of Voretigene Neparvovec Treatment for Biallelic RPE65-Mediated Inherited Retinal Dystrophies in a Cohort of Patients from a Single Center.
Kiraly P, Cottriall CL, Taylor LJ, Jolly JK, Cehajic-Kapetanovic J, Yusuf IH, Martinez-Fernandez de la Camara C, Shanks M, Downes SM, MacLaren RE, Fischer MD. Kiraly P, et al. Among authors: martinez fernandez de la camara c. Biomolecules. 2023 Oct 5;13(10):1484. doi: 10.3390/biom13101484. Biomolecules. 2023. PMID: 37892166 Free PMC article.
Impaired glutamylation of RPGRORF15 underlies the cone-dominated phenotype associated with truncating distal ORF15 variants.
Cehajic-Kapetanovic J, Martinez-Fernandez de la Camara C, Birtel J, Rehman S, McClements ME, Charbel Issa P, Lotery AJ, MacLaren RE. Cehajic-Kapetanovic J, et al. Among authors: martinez fernandez de la camara c. Proc Natl Acad Sci U S A. 2022 Dec 6;119(49):e2208707119. doi: 10.1073/pnas.2208707119. Epub 2022 Nov 29. Proc Natl Acad Sci U S A. 2022. PMID: 36445968 Free PMC article.
25 results