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Rationally designed AAV2 and AAVrh8R capsids provide improved transduction in the retina and brain.
Gene Ther. 2018 Jun;25(3):205-219. doi: 10.1038/s41434-018-0017-8. Epub 2018 May 22.
Gene Ther. 2018.
PMID: 29785047
Genetic modifiers of liver disease in cystic fibrosis.
Bartlett JR, Friedman KJ, Ling SC, Pace RG, Bell SC, Bourke B, Castaldo G, Castellani C, Cipolli M, Colombo C, Colombo JL, Debray D, Fernandez A, Lacaille F, Macek M Jr, Rowland M, Salvatore F, Taylor CJ, Wainwright C, Wilschanski M, Zemková D, Hannah WB, Phillips MJ, Corey M, Zielenski J, Dorfman R, Wang Y, Zou F, Silverman LM, Drumm ML, Wright FA, Lange EM, Durie PR, Knowles MR; Gene Modifier Study Group.
Bartlett JR, et al.
JAMA. 2009 Sep 9;302(10):1076-83. doi: 10.1001/jama.2009.1295.
JAMA. 2009.
PMID: 19738092
Free PMC article.
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