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Autor:
Mark J. Osborn, Ron T. McElmurry, Gordon J. Freeman, Bruce R. Blazar, Anthony P. DeFeo, Zhi-Ying Chen, Christopher J. Lees, Jakub Tolar, Mark A. Kay, Luigi Naldini
Publikováno v:
Molecular Therapy. 19(3):450-460
Mucopolysaccharidosis type I (MPS I) is a lysosomal storage disease characterized by mutations to the α-L-iduronidase (IDUA) gene resulting in inactivation of the IDUA enzyme. The loss of IDUA protein results in the progressive accumulation of glyco
Autor:
Webber BR; Department of Pediatrics, University of Minnesota, Minneapolis, MN, USA. webb0178@umn.edu.; Masonic Cancer Center, University of Minnesota, Minneapolis, MN, USA. webb0178@umn.edu.; Center for Genome Engineering, University of Minnesota, Minneapolis, MN, USA. webb0178@umn.edu., Johnson MJ; Department of Pediatrics, University of Minnesota, Minneapolis, MN, USA.; Masonic Cancer Center, University of Minnesota, Minneapolis, MN, USA.; Center for Genome Engineering, University of Minnesota, Minneapolis, MN, USA., Skeate JG; Department of Pediatrics, University of Minnesota, Minneapolis, MN, USA.; Masonic Cancer Center, University of Minnesota, Minneapolis, MN, USA.; Center for Genome Engineering, University of Minnesota, Minneapolis, MN, USA., Slipek NJ; Department of Pediatrics, University of Minnesota, Minneapolis, MN, USA.; Masonic Cancer Center, University of Minnesota, Minneapolis, MN, USA.; Center for Genome Engineering, University of Minnesota, Minneapolis, MN, USA., Lahr WS; Department of Pediatrics, University of Minnesota, Minneapolis, MN, USA.; Masonic Cancer Center, University of Minnesota, Minneapolis, MN, USA.; Center for Genome Engineering, University of Minnesota, Minneapolis, MN, USA., DeFeo AP; Department of Pediatrics, University of Minnesota, Minneapolis, MN, USA.; Masonic Cancer Center, University of Minnesota, Minneapolis, MN, USA.; Center for Genome Engineering, University of Minnesota, Minneapolis, MN, USA., Mills LJ; Department of Pediatrics, University of Minnesota, Minneapolis, MN, USA.; Masonic Cancer Center, University of Minnesota, Minneapolis, MN, USA., Qiu X; Department of Pediatrics, University of Minnesota, Minneapolis, MN, USA.; Masonic Cancer Center, University of Minnesota, Minneapolis, MN, USA.; Center for Genome Engineering, University of Minnesota, Minneapolis, MN, USA., Rathmann B; Department of Pediatrics, University of Minnesota, Minneapolis, MN, USA.; Masonic Cancer Center, University of Minnesota, Minneapolis, MN, USA.; Center for Genome Engineering, University of Minnesota, Minneapolis, MN, USA., Diers MD; Department of Pediatrics, University of Minnesota, Minneapolis, MN, USA.; Masonic Cancer Center, University of Minnesota, Minneapolis, MN, USA.; Center for Genome Engineering, University of Minnesota, Minneapolis, MN, USA., Wick B; Department of Pediatrics, University of Minnesota, Minneapolis, MN, USA.; Masonic Cancer Center, University of Minnesota, Minneapolis, MN, USA.; Center for Genome Engineering, University of Minnesota, Minneapolis, MN, USA., Henley T; Intima Bioscience, New York, NY, USA., Choudhry M; Intima Bioscience, New York, NY, USA., Starr TK; Masonic Cancer Center, University of Minnesota, Minneapolis, MN, USA.; Department of Genetics, Cell Biology and Development, University of Minnesota, Minneapolis, MN, USA.; Department of Ob-Gyn and Women's Health, University of Minnesota, Minneapolis, MN, USA., McIvor RS; Center for Genome Engineering, University of Minnesota, Minneapolis, MN, USA.; Department of Genetics, Cell Biology and Development, University of Minnesota, Minneapolis, MN, USA., Moriarity BS; Department of Pediatrics, University of Minnesota, Minneapolis, MN, USA. mori0164@umn.edu.; Masonic Cancer Center, University of Minnesota, Minneapolis, MN, USA. mori0164@umn.edu.; Center for Genome Engineering, University of Minnesota, Minneapolis, MN, USA. mori0164@umn.edu.
Publikováno v:
Nature biomedical engineering [Nat Biomed Eng] 2023 Dec 13. Date of Electronic Publication: 2023 Dec 13.
Autor:
Sipe CJ; Department of Pediatrics, University of Minnesota, Minneapolis, MN 55455, USA.; Masonic Cancer Center, University of Minnesota, Minneapolis, MN 55455, USA.; Center for Genome Engineering, University of Minnesota, Minneapolis, MN 55455, USA.; Stem Cell Institute, University of Minnesota, Minneapolis, MN 55455, USA., Kluesner MG; Department of Pediatrics, University of Minnesota, Minneapolis, MN 55455, USA.; Masonic Cancer Center, University of Minnesota, Minneapolis, MN 55455, USA.; Center for Genome Engineering, University of Minnesota, Minneapolis, MN 55455, USA.; Stem Cell Institute, University of Minnesota, Minneapolis, MN 55455, USA.; Medical Scientist Training Program, University of Washington, Seattle, WA 98195, USA., Bingea SP; Department of Pediatrics, University of Minnesota, Minneapolis, MN 55455, USA.; Masonic Cancer Center, University of Minnesota, Minneapolis, MN 55455, USA.; Center for Genome Engineering, University of Minnesota, Minneapolis, MN 55455, USA.; Stem Cell Institute, University of Minnesota, Minneapolis, MN 55455, USA., Lahr WS; Department of Pediatrics, University of Minnesota, Minneapolis, MN 55455, USA.; Masonic Cancer Center, University of Minnesota, Minneapolis, MN 55455, USA.; Center for Genome Engineering, University of Minnesota, Minneapolis, MN 55455, USA.; Stem Cell Institute, University of Minnesota, Minneapolis, MN 55455, USA., Andrew AA; Department of Pediatrics, University of Minnesota, Minneapolis, MN 55455, USA.; Masonic Cancer Center, University of Minnesota, Minneapolis, MN 55455, USA.; Center for Genome Engineering, University of Minnesota, Minneapolis, MN 55455, USA.; Stem Cell Institute, University of Minnesota, Minneapolis, MN 55455, USA., Wang M; Department of Pediatrics, University of Minnesota, Minneapolis, MN 55455, USA.; Masonic Cancer Center, University of Minnesota, Minneapolis, MN 55455, USA.; Center for Genome Engineering, University of Minnesota, Minneapolis, MN 55455, USA.; Stem Cell Institute, University of Minnesota, Minneapolis, MN 55455, USA., DeFeo AP; Department of Pediatrics, University of Minnesota, Minneapolis, MN 55455, USA.; Masonic Cancer Center, University of Minnesota, Minneapolis, MN 55455, USA.; Center for Genome Engineering, University of Minnesota, Minneapolis, MN 55455, USA.; Stem Cell Institute, University of Minnesota, Minneapolis, MN 55455, USA., Hinkel TL; Department of Pediatrics, University of Minnesota, Minneapolis, MN 55455, USA.; Masonic Cancer Center, University of Minnesota, Minneapolis, MN 55455, USA.; Center for Genome Engineering, University of Minnesota, Minneapolis, MN 55455, USA.; Stem Cell Institute, University of Minnesota, Minneapolis, MN 55455, USA., Laoharawee K; Department of Pediatrics, University of Minnesota, Minneapolis, MN 55455, USA.; Masonic Cancer Center, University of Minnesota, Minneapolis, MN 55455, USA.; Center for Genome Engineering, University of Minnesota, Minneapolis, MN 55455, USA.; Stem Cell Institute, University of Minnesota, Minneapolis, MN 55455, USA., Wagner JE; Department of Pediatrics, University of Minnesota, Minneapolis, MN 55455, USA.; Division of Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN 55455, USA., MacMillan ML; Department of Pediatrics, University of Minnesota, Minneapolis, MN 55455, USA.; Division of Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN 55455, USA., Vercellotti GM; Division of Hematology, Oncology and Transplantation, University of Minnesota, Minneapolis, MN 55455, USA., Tolar J; Department of Pediatrics, University of Minnesota, Minneapolis, MN 55455, USA.; Stem Cell Institute, University of Minnesota, Minneapolis, MN 55455, USA.; Division of Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN 55455, USA., Osborn MJ; Department of Pediatrics, University of Minnesota, Minneapolis, MN 55455, USA.; Division of Hematology, Oncology and Transplantation, University of Minnesota, Minneapolis, MN 55455, USA., McIvor RS; Department of Pediatrics, University of Minnesota, Minneapolis, MN 55455, USA.; Masonic Cancer Center, University of Minnesota, Minneapolis, MN 55455, USA.; Center for Genome Engineering, University of Minnesota, Minneapolis, MN 55455, USA.; Stem Cell Institute, University of Minnesota, Minneapolis, MN 55455, USA., Webber BR; Department of Pediatrics, University of Minnesota, Minneapolis, MN 55455, USA.; Masonic Cancer Center, University of Minnesota, Minneapolis, MN 55455, USA.; Center for Genome Engineering, University of Minnesota, Minneapolis, MN 55455, USA.; Stem Cell Institute, University of Minnesota, Minneapolis, MN 55455, USA., Moriarity BS; Department of Pediatrics, University of Minnesota, Minneapolis, MN 55455, USA.; Masonic Cancer Center, University of Minnesota, Minneapolis, MN 55455, USA.; Center for Genome Engineering, University of Minnesota, Minneapolis, MN 55455, USA.; Stem Cell Institute, University of Minnesota, Minneapolis, MN 55455, USA.
Publikováno v:
International journal of molecular sciences [Int J Mol Sci] 2022 Jul 29; Vol. 23 (15). Date of Electronic Publication: 2022 Jul 29.
Autor:
Skvarova Kramarzova K; Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN 55455, USA. karolina.skvarova@gmail.com.; Childhood Leukemia Investigation Prague (CLIP), Department of Pediatric Hematology and Oncology, Second Faculty of Medicine, Charles University, Prague 15006, Czech Republic. karolina.skvarova@gmail.com., Osborn MJ; Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN 55455, USA. osbor026@umn.edu.; Stem Cell Institute, University of Minnesota, Minneapolis, MN 55455, USA. osbor026@umn.edu.; Center for Genome Engineering, University of Minnesota, Minneapolis, MN 55455, USA. osbor026@umn.edu.; Asan-Minnesota Institute for Innovating Transplantation, University of Minnesota, Minneapolis, MN 55455, USA. osbor026@umn.edu., Webber BR; Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN 55455, USA. webb0178@umn.edu., DeFeo AP; Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN 55455, USA. apdefeo@umn.edu., McElroy AN; Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN 55455, USA. leira001@umn.edu., Kim CJ; Asan Institute for Life Sciences, Asan Medical Center, Asan-Minnesota Institute for Innovating Transplantation, Seoul 138-736, Korea. ckim@amc.seoul.kr., Tolar J; Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN 55455, USA. tolar003@umn.edu.; Stem Cell Institute, University of Minnesota, Minneapolis, MN 55455, USA. tolar003@umn.edu.; Asan-Minnesota Institute for Innovating Transplantation, University of Minnesota, Minneapolis, MN 55455, USA. tolar003@umn.edu.
Publikováno v:
International journal of molecular sciences [Int J Mol Sci] 2017 Jun 14; Vol. 18 (6). Date of Electronic Publication: 2017 Jun 14.
Autor:
Osborn MJ; Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, 420 Delaware ST SE, MMC 366, Minneapolis, MN, 55455, USA. osbor026@umn.edu.; Center for Genome Engineering, University of Minnesota, Minneapolis, MN, USA. osbor026@umn.edu.; Stem Cell Institute, University of Minnesota, Minneapolis, MN, USA. osbor026@umn.edu.; Asan-Minnesota Institute for Innovating Transplantation, Seoul, Republic of Korea. osbor026@umn.edu.; School of Public Health, University of Minnesota, Minneapolis, MN, USA. osbor026@umn.edu., Webber BR; Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, 420 Delaware ST SE, MMC 366, Minneapolis, MN, 55455, USA., McElmurry RT; Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, 420 Delaware ST SE, MMC 366, Minneapolis, MN, 55455, USA., Rudser KD; Department of Cell Biology and Anatomy and the Alberta Children's Hospital Research Institute, University of Calgary, Calgary, AB, Canada., DeFeo AP; Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, 420 Delaware ST SE, MMC 366, Minneapolis, MN, 55455, USA., Muradian M; Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, 420 Delaware ST SE, MMC 366, Minneapolis, MN, 55455, USA., Petryk A; Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, 420 Delaware ST SE, MMC 366, Minneapolis, MN, 55455, USA., Hallgrimsson B; Department of Cell Biology and Anatomy and the Alberta Children's Hospital Research Institute, University of Calgary, Calgary, AB, Canada., Blazar BR; Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, 420 Delaware ST SE, MMC 366, Minneapolis, MN, 55455, USA., Tolar J; Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, 420 Delaware ST SE, MMC 366, Minneapolis, MN, 55455, USA.; Stem Cell Institute, University of Minnesota, Minneapolis, MN, USA.; Asan-Minnesota Institute for Innovating Transplantation, Seoul, Republic of Korea.; School of Public Health, University of Minnesota, Minneapolis, MN, USA., Braunlin EA; Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, 420 Delaware ST SE, MMC 366, Minneapolis, MN, 55455, USA. braun002@umn.edu.; Lillehei Heart Institute, University of Minnesota, Minneapolis, MN, USA. braun002@umn.edu.
Publikováno v:
Journal of inherited metabolic disease [J Inherit Metab Dis] 2017 Mar; Vol. 40 (2), pp. 281-289. Date of Electronic Publication: 2016 Oct 14.
Autor:
Osborn M; University of Minnesota Twin Cities, 5635, Pediatrics, Minneapolis, Minnesota, United States.; University of Minnesota Center for Genome Engineering, 189708, Minneapolis, Minnesota, United States.; Stem Cell Institute, Minneapolis, Minnesota, United States ; osbor026@umn.edu., Lonetree CL; University of Minnesota Twin Cities, 5635, Pediatrics, Minneapolis, Minnesota, United States ; lonet005@umn.edu., Webber BR; University of Minnesota Twin Cities, 5635, Pediatrics, Minneapolis, Minnesota, United States ; webb0178@umn.edu., Patel D; University of Minnesota Twin Cities, 5635, Pediatrics, Minneapolis, Minnesota, United States ; ddpatel@umn.edu., Dunmire S; University of Minnesota Twin Cities, 5635, Pediatrics, Minneapolis, Minnesota, United States ; dunmi002@umn.edu., McElroy AN; University of Minnesota Twin Cities, 5635, Pediatrics, Minneapolis, Minnesota, United States ; leira001@umn.edu., DeFeo AP; University of Minnesota Twin Cities, 5635, Pediatrics, Minneapolis, Minnesota, United States ; apdefeo@umn.edu., MacMillan ML; University of Minnesota Twin Cities, 5635, Pediatrics, Minneapolis, Minnesota, United States ; macmi002@umn.edu., Wagner J; University of Minnesota Twin Cities, 5635, Pediatrics, Minneapolis, Minnesota, United States ; wagne002@umn.edu., Balzar BR; University of Minnesota Twin Cities, 5635, Pediatrics, Minneapolis, Minnesota, United States ; blaza001@umn.edu., Tolar J; University of Minnesota Twin Cities, 5635, Pediatrics, Minneapolis, Minnesota, United States.; Stem Cell Institute, Minneapolis, Minnesota, United States ; tolar003@umn.edu.
Publikováno v:
Stem cells and development [Stem Cells Dev] 2016 Oct; Vol. 25 (20), pp. 1591-1603. Date of Electronic Publication: 2016 Aug 18.
Autor:
Osborn MJ; Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, Minneapolis, Minnesota, USA.; Center for Genome Engineering, University of Minnesota, Minneapolis, Minnesota, USA.; Stem Cell Institute, University of Minnesota, Minneapolis, Minnesota, USA., Webber BR; Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, Minneapolis, Minnesota, USA., Knipping F; German Cancer Research Center (DKFZ), Heidelberg, Germany.; Department of Translational Oncology, National Center for Tumor Diseases, Heidelberg, Germany., Lonetree CL; Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, Minneapolis, Minnesota, USA., Tennis N; Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, Minneapolis, Minnesota, USA., DeFeo AP; Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, Minneapolis, Minnesota, USA., McElroy AN; Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, Minneapolis, Minnesota, USA., Starker CG; Center for Genome Engineering, University of Minnesota, Minneapolis, Minnesota, USA.; Ben Towne Center for Childhood Cancer Research, Seattle Children's Research Institute, Seattle, Washington, USA., Lee C; Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, Minneapolis, Minnesota, USA., Merkel S; Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, Minneapolis, Minnesota, USA., Lund TC; Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, Minneapolis, Minnesota, USA., Kelly-Spratt KS; Ben Towne Center for Childhood Cancer Research, Seattle Children's Research Institute, Seattle, Washington, USA., Jensen MC; Ben Towne Center for Childhood Cancer Research, Seattle Children's Research Institute, Seattle, Washington, USA.; Department of Pediatrics, University of Washington, Seattle, Washington, USA., Voytas DF; Center for Genome Engineering, University of Minnesota, Minneapolis, Minnesota, USA.; Department of Genetics, Cell Biology and Development, University of Minnesota, Minneapolis, Minnesota, USA., von Kalle C; German Cancer Research Center (DKFZ), Heidelberg, Germany.; Department of Translational Oncology, National Center for Tumor Diseases, Heidelberg, Germany., Schmidt M; German Cancer Research Center (DKFZ), Heidelberg, Germany.; Department of Translational Oncology, National Center for Tumor Diseases, Heidelberg, Germany., Gabriel R; German Cancer Research Center (DKFZ), Heidelberg, Germany.; Department of Translational Oncology, National Center for Tumor Diseases, Heidelberg, Germany., Hippen KL; Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, Minneapolis, Minnesota, USA., Miller JS; Department of Medicine, University of Minnesota, Minneapolis, Minnesota, USA., Scharenberg AM; Seattle Children's Research Institute, and University of Washington School of Medicine, Seattle, Washington, USA., Tolar J; Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, Minneapolis, Minnesota, USA.; Stem Cell Institute, University of Minnesota, Minneapolis, Minnesota, USA., Blazar BR; Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, Minneapolis, Minnesota, USA.
Publikováno v:
Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2016 Mar; Vol. 24 (3), pp. 570-81. Date of Electronic Publication: 2015 Oct 27.
Autor:
Webber BR; Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN, USA., Osborn MJ; Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN, USA.; Stem Cell Institute, University of Minnesota, Minneapolis, MN, USA.; Center for Genome Engineering, University of Minnesota, Minneapolis, MN, USA.; Asan-Minnesota Institute for Innovating Transplantation, Seoul, Republic of Korea., McElroy AN; Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN, USA., Twaroski K; Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN, USA., Lonetree CL; Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN, USA., DeFeo AP; Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN, USA., Xia L; Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN, USA., Eide C; Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN, USA., Lees CJ; Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN, USA., McElmurry RT; Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN, USA., Riddle MJ; Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN, USA., Kim CJ; Asan-Minnesota Institute for Innovating Transplantation, Seoul, Republic of Korea., Patel DD; Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN, USA., Blazar BR; Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN, USA.; Stem Cell Institute, University of Minnesota, Minneapolis, MN, USA., Tolar J; Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN, USA.; Stem Cell Institute, University of Minnesota, Minneapolis, MN, USA.; Asan-Minnesota Institute for Innovating Transplantation, Seoul, Republic of Korea.
Publikováno v:
NPJ Regenerative medicine [NPJ Regen Med] 2016; Vol. 1. Date of Electronic Publication: 2016 Dec 08.
Autor:
Osborn MJ; 1 Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota , Minneapolis, MN 55455., 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
Publikováno v:
Human gene therapy [Hum Gene Ther] 2015 Feb; Vol. 26 (2), pp. 114-26.