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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. Among authors: schmidt m. 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. Among authors: schmidt m. 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.
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: schmidt m. Hum Gene Ther. 2015 Feb;26(2):114-26. doi: 10.1089/hum.2014.111. Hum Gene Ther. 2015. PMID: 25545896 Free PMC article.
Successful engraftment of gene-corrected hematopoietic stem cells in non-conditioned patients with Fanconi anemia.
Río P, Navarro S, Wang W, Sánchez-Domínguez R, Pujol RM, Segovia JC, Bogliolo M, Merino E, Wu N, Salgado R, Lamana ML, Yañez RM, Casado JA, Giménez Y, Román-Rodríguez FJ, Álvarez L, Alberquilla O, Raimbault A, Guenechea G, Lozano ML, Cerrato L, Hernando M, Gálvez E, Hladun R, Giralt I, Barquinero J, Galy A, García de Andoín N, López R, Catalá A, Schwartz JD, Surrallés J, Soulier J, Schmidt M, Díaz de Heredia C, Sevilla J, Bueren JA. Río P, et al. Among authors: schmidt m. Nat Med. 2019 Sep;25(9):1396-1401. doi: 10.1038/s41591-019-0550-z. Epub 2019 Sep 9. Nat Med. 2019. PMID: 31501599 Clinical Trial.
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: schmidt m. 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.
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: schmidt m. 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.
Sleeping beauty transposition from nonintegrating lentivirus.
Vink CA, Gaspar HB, Gabriel R, Schmidt M, McIvor RS, Thrasher AJ, Qasim W. Vink CA, et al. Among authors: schmidt m. Mol Ther. 2009 Jul;17(7):1197-204. doi: 10.1038/mt.2009.94. Epub 2009 May 5. Mol Ther. 2009. PMID: 19417741 Free PMC article.
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