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Assessment of AAV Vector Tropisms for Mouse and Human Pluripotent Stem Cell-Derived RPE and Photoreceptor Cells.
Gonzalez-Cordero A, Goh D, Kruczek K, Naeem A, Fernando M, Kleine Holthaus SM, Takaaki M, Blackford SJI, Kloc M, Agundez L, Sampson RD, Borooah S, Ovando-Roche P, Mehat MS, West EL, Smith AJ, Pearson RA, Ali RR. Gonzalez-Cordero A, et al. Among authors: kleine holthaus sm. Hum Gene Ther. 2018 Oct;29(10):1124-1139. doi: 10.1089/hum.2018.027. Epub 2018 May 14. Hum Gene Ther. 2018. PMID: 29580100
Prevention of Photoreceptor Cell Loss in a Cln6nclf Mouse Model of Batten Disease Requires CLN6 Gene Transfer to Bipolar Cells.
Kleine Holthaus SM, Ribeiro J, Abelleira-Hervas L, Pearson RA, Duran Y, Georgiadis A, Sampson RD, Rizzi M, Hoke J, Maswood R, Azam S, Luhmann UFO, Smith AJ, Mole SE, Ali RR. Kleine Holthaus SM, et al. Mol Ther. 2018 May 2;26(5):1343-1353. doi: 10.1016/j.ymthe.2018.02.027. Epub 2018 Mar 2. Mol Ther. 2018. PMID: 29606505 Free PMC article.
Experimental gene therapies for the NCLs.
Liu W, Kleine-Holthaus SM, Herranz-Martin S, Aristorena M, Mole SE, Smith AJ, Ali RR, Rahim AA. Liu W, et al. Among authors: kleine holthaus sm. Biochim Biophys Acta Mol Basis Dis. 2020 Sep 1;1866(9):165772. doi: 10.1016/j.bbadis.2020.165772. Epub 2020 Mar 24. Biochim Biophys Acta Mol Basis Dis. 2020. PMID: 32220628 Free article. Review.