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Coupling endonucleases with DNA end-processing enzymes to drive gene disruption.
Certo MT, Gwiazda KS, Kuhar R, Sather B, Curinga G, Mandt T, Brault M, Lambert AR, Baxter SK, Jacoby K, Ryu BY, Kiem HP, Gouble A, Paques F, Rawlings DJ, Scharenberg AM. Certo MT, et al. Among authors: rawlings dj. Nat Methods. 2012 Oct;9(10):973-5. doi: 10.1038/nmeth.2177. Epub 2012 Sep 2. Nat Methods. 2012. PMID: 22941364 Free PMC article.
Intravenous injection of a foamy virus vector to correct canine SCID-X1.
Burtner CR, Beard BC, Kennedy DR, Wohlfahrt ME, Adair JE, Trobridge GD, Scharenberg AM, Torgerson TR, Rawlings DJ, Felsburg PJ, Kiem HP. Burtner CR, et al. Among authors: rawlings dj. Blood. 2014 Jun 5;123(23):3578-84. doi: 10.1182/blood-2013-11-538926. Epub 2014 Mar 18. Blood. 2014. PMID: 24642749 Free PMC article.
Rapid immune reconstitution of SCID-X1 canines after G-CSF/AMD3100 mobilization and in vivo gene therapy.
Humbert O, Chan F, Rajawat YS, Torgerson TR, Burtner CR, Hubbard NW, Humphrys D, Norgaard ZK, O'Donnell P, Adair JE, Trobridge GD, Scharenberg AM, Felsburg PJ, Rawlings DJ, Kiem HP. Humbert O, et al. Among authors: rawlings dj. Blood Adv. 2018 May 8;2(9):987-999. doi: 10.1182/bloodadvances.2018016451. Blood Adv. 2018. PMID: 29720491 Free PMC article.
TALEN-Mediated Gene Editing of HBG in Human Hematopoietic Stem Cells Leads to Therapeutic Fetal Hemoglobin Induction.
Lux CT, Pattabhi S, Berger M, Nourigat C, Flowers DA, Negre O, Humbert O, Yang JG, Lee C, Jacoby K, Bernstein I, Kiem HP, Scharenberg A, Rawlings DJ. Lux CT, et al. Among authors: rawlings dj. Mol Ther Methods Clin Dev. 2018 Dec 31;12:175-183. doi: 10.1016/j.omtm.2018.12.008. eCollection 2019 Mar 15. Mol Ther Methods Clin Dev. 2018. PMID: 30705922 Free PMC article.
257 results