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Gene therapy for Fanconi anemia: one step closer to the clinic.
Tolar J, Becker PS, Clapp DW, Hanenberg H, de Heredia CD, Kiem HP, Navarro S, Qasba P, Rio P, Schmidt M, Sevilla J, Verhoeyen E, Thrasher AJ, Bueren J. Tolar J, et al. Hum Gene Ther. 2012 Feb;23(2):141-4. doi: 10.1089/hum.2011.237. Hum Gene Ther. 2012. PMID: 22248350 Free PMC article. No abstract available.
Stem cell gene therapy for fanconi anemia: report from the 1st international Fanconi anemia gene therapy working group meeting.
Tolar J, Adair JE, Antoniou M, Bartholomae CC, Becker PS, Blazar BR, Bueren J, Carroll T, Cavazzana-Calvo M, Clapp DW, Dalgleish R, Galy A, Gaspar HB, Hanenberg H, Von Kalle C, Kiem HP, Lindeman D, Naldini L, Navarro S, Renella R, Rio P, Sevilla J, Schmidt M, Verhoeyen E, Wagner JE, Williams DA, Thrasher AJ. Tolar J, et al. Mol Ther. 2011 Jul;19(7):1193-8. doi: 10.1038/mt.2011.78. Epub 2011 May 3. Mol Ther. 2011. PMID: 21540837 Free PMC article. Review.
Synthetic zinc finger nuclease design and rapid assembly.
Osborn MJ, DeFeo AP, Blazar BR, Tolar J. Osborn MJ, et al. Among authors: tolar j. Hum Gene Ther. 2011 Sep;22(9):1155-65. doi: 10.1089/hum.2011.072. Epub 2011 Aug 10. Hum Gene Ther. 2011. PMID: 21663559 Free PMC article.
Sleeping Beauty-mediated correction of Fanconi anemia type C.
Hyland KA, Olson ER, Clark KJ, Aronovich EL, Hackett PB, Blazar BR, Tolar J, Scott McIvor R. Hyland KA, et al. Among authors: tolar j. J Gene Med. 2011 Sep;13(9):462-9. doi: 10.1002/jgm.1589. J Gene Med. 2011. PMID: 21766398
TALEN-based gene correction for epidermolysis bullosa.
Osborn MJ, Starker CG, McElroy AN, Webber BR, Riddle MJ, Xia L, DeFeo AP, Gabriel R, Schmidt M, von Kalle C, Carlson DF, Maeder ML, Joung JK, Wagner JE, Voytas DF, Blazar BR, Tolar J. Osborn MJ, et al. Among authors: tolar j. Mol Ther. 2013 Jun;21(6):1151-9. doi: 10.1038/mt.2013.56. Epub 2013 Apr 2. Mol Ther. 2013. PMID: 23546300 Free PMC article.
Fanconi anemia gene editing by the CRISPR/Cas9 system.
Osborn MJ, Gabriel R, Webber BR, DeFeo AP, McElroy AN, Jarjour J, Starker CG, Wagner JE, Joung JK, Voytas DF, von Kalle C, Schmidt M, Blazar BR, Tolar J. Osborn MJ, et al. Among authors: tolar j. Hum Gene Ther. 2015 Feb;26(2):114-26. doi: 10.1089/hum.2014.111. Hum Gene Ther. 2015. PMID: 25545896 Free PMC article.
Evaluation of TCR Gene Editing Achieved by TALENs, CRISPR/Cas9, and megaTAL Nucleases.
Osborn MJ, Webber BR, Knipping F, Lonetree CL, Tennis N, DeFeo AP, McElroy AN, Starker CG, Lee C, Merkel S, Lund TC, Kelly-Spratt KS, Jensen MC, Voytas DF, von Kalle C, Schmidt M, Gabriel R, Hippen KL, Miller JS, Scharenberg AM, Tolar J, Blazar BR. Osborn MJ, et al. Among authors: tolar j. Mol Ther. 2016 Mar;24(3):570-81. doi: 10.1038/mt.2015.197. Epub 2015 Oct 27. Mol Ther. 2016. PMID: 26502778 Free PMC article.
272 results