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Page 1
Targeted gene correction of α1-antitrypsin deficiency in induced pluripotent stem cells.
Yusa K, Rashid ST, Strick-Marchand H, Varela I, Liu PQ, Paschon DE, Miranda E, Ordóñez A, Hannan NR, Rouhani FJ, Darche S, Alexander G, Marciniak SJ, Fusaki N, Hasegawa M, Holmes MC, Di Santo JP, Lomas DA, Bradley A, Vallier L. Yusa K, et al. Among authors: paschon de. Nature. 2011 Oct 12;478(7369):391-4. doi: 10.1038/nature10424. Nature. 2011. PMID: 21993621 Free PMC article.
In vivo genome editing restores haemostasis in a mouse model of haemophilia.
Li H, Haurigot V, Doyon Y, Li T, Wong SY, Bhagwat AS, Malani N, Anguela XM, Sharma R, Ivanciu L, Murphy SL, Finn JD, Khazi FR, Zhou S, Paschon DE, Rebar EJ, Bushman FD, Gregory PD, Holmes MC, High KA. Li H, et al. Among authors: paschon de. Nature. 2011 Jun 26;475(7355):217-21. doi: 10.1038/nature10177. Nature. 2011. PMID: 21706032 Free PMC article.
Targeted correction and restored function of the CFTR gene in cystic fibrosis induced pluripotent stem cells.
Crane AM, Kramer P, Bui JH, Chung WJ, Li XS, Gonzalez-Garay ML, Hawkins F, Liao W, Mora D, Choi S, Wang J, Sun HC, Paschon DE, Guschin DY, Gregory PD, Kotton DN, Holmes MC, Sorscher EJ, Davis BR. Crane AM, et al. Among authors: paschon de. Stem Cell Reports. 2015 Apr 14;4(4):569-77. doi: 10.1016/j.stemcr.2015.02.005. Epub 2015 Mar 12. Stem Cell Reports. 2015. PMID: 25772471 Free PMC article.
Functional footprinting of regulatory DNA.
Vierstra J, Reik A, Chang KH, Stehling-Sun S, Zhou Y, Hinkley SJ, Paschon DE, Zhang L, Psatha N, Bendana YR, O'Neil CM, Song AH, Mich AK, Liu PQ, Lee G, Bauer DE, Holmes MC, Orkin SH, Papayannopoulou T, Stamatoyannopoulos G, Rebar EJ, Gregory PD, Urnov FD, Stamatoyannopoulos JA. Vierstra J, et al. Among authors: paschon de. Nat Methods. 2015 Oct;12(10):927-30. doi: 10.1038/nmeth.3554. Epub 2015 Aug 31. Nat Methods. 2015. PMID: 26322838 Free PMC article.
Targeted gene addition in human CD34(+) hematopoietic cells for correction of X-linked chronic granulomatous disease.
De Ravin SS, Reik A, Liu PQ, Li L, Wu X, Su L, Raley C, Theobald N, Choi U, Song AH, Chan A, Pearl JR, Paschon DE, Lee J, Newcombe H, Koontz S, Sweeney C, Shivak DA, Zarember KA, Peshwa MV, Gregory PD, Urnov FD, Malech HL. De Ravin SS, et al. Among authors: paschon de. Nat Biotechnol. 2016 Apr;34(4):424-9. doi: 10.1038/nbt.3513. Epub 2016 Mar 7. Nat Biotechnol. 2016. PMID: 26950749 Free PMC article.
Editing T cell specificity towards leukemia by zinc finger nucleases and lentiviral gene transfer.
Provasi E, Genovese P, Lombardo A, Magnani Z, Liu PQ, Reik A, Chu V, Paschon DE, Zhang L, Kuball J, Camisa B, Bondanza A, Casorati G, Ponzoni M, Ciceri F, Bordignon C, Greenberg PD, Holmes MC, Gregory PD, Naldini L, Bonini C. Provasi E, et al. Among authors: paschon de. Nat Med. 2012 May;18(5):807-815. doi: 10.1038/nm.2700. Nat Med. 2012. PMID: 22466705 Free PMC article.
Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells.
Hoban MD, Cost GJ, Mendel MC, Romero Z, Kaufman ML, Joglekar AV, Ho M, Lumaquin D, Gray D, Lill GR, Cooper AR, Urbinati F, Senadheera S, Zhu A, Liu PQ, Paschon DE, Zhang L, Rebar EJ, Wilber A, Wang X, Gregory PD, Holmes MC, Reik A, Hollis RP, Kohn DB. Hoban MD, et al. Among authors: paschon de. Blood. 2015 Apr 23;125(17):2597-604. doi: 10.1182/blood-2014-12-615948. Epub 2015 Mar 2. Blood. 2015. PMID: 25733580 Free PMC article.
A TALE nuclease architecture for efficient genome editing.
Miller JC, Tan S, Qiao G, Barlow KA, Wang J, Xia DF, Meng X, Paschon DE, Leung E, Hinkley SJ, Dulay GP, Hua KL, Ankoudinova I, Cost GJ, Urnov FD, Zhang HS, Holmes MC, Zhang L, Gregory PD, Rebar EJ. Miller JC, et al. Among authors: paschon de. Nat Biotechnol. 2011 Feb;29(2):143-8. doi: 10.1038/nbt.1755. Epub 2010 Dec 22. Nat Biotechnol. 2011. PMID: 21179091
Diversifying the structure of zinc finger nucleases for high-precision genome editing.
Paschon DE, Lussier S, Wangzor T, Xia DF, Li PW, Hinkley SJ, Scarlott NA, Lam SC, Waite AJ, Truong LN, Gandhi N, Kadam BN, Patil DP, Shivak DA, Lee GK, Holmes MC, Zhang L, Miller JC, Rebar EJ. Paschon DE, et al. Nat Commun. 2019 Mar 8;10(1):1133. doi: 10.1038/s41467-019-08867-x. Nat Commun. 2019. PMID: 30850604 Free PMC article.
Gene Correction of iPSCs from a Wiskott-Aldrich Syndrome Patient Normalizes the Lymphoid Developmental and Functional Defects.
Laskowski TJ, Van Caeneghem Y, Pourebrahim R, Ma C, Ni Z, Garate Z, Crane AM, Li XS, Liao W, Gonzalez-Garay M, Segovia JC, Paschon DE, Rebar EJ, Holmes MC, Kaufman D, Vandekerckhove B, Davis BR. Laskowski TJ, et al. Among authors: paschon de. Stem Cell Reports. 2016 Aug 9;7(2):139-48. doi: 10.1016/j.stemcr.2016.06.003. Epub 2016 Jul 7. Stem Cell Reports. 2016. PMID: 27396937 Free PMC article.
26 results