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| OPPOSITION | - Pan Dao, Gunther Roland, Duan Weiming, Wendell Steve, Kaemmerer William, Kafri Tal, Verma Inder M., Whitley Chester B., "Biodistribution and Toxicity Studies of VSVG-Pseudotyped Lentiviral Vector after Intravenous Administration in Mice with the Observation of in Vivo Transduction of Bone Marrow", Molecular Therapy, Elsevier Inc., US, US , (20020701), vol. 6, no. 1, doi:10.1006/mthe.2002.0630, ISSN 1525-0016, pages 19 - 29, XP093210530 | |
| OPPOSITION | - Harrison Brown, "Target-Cell-Directed Bioengineering Approaches for Gene Therapy of Hemophilia A", Molecular Therapy: Methods & Clinical Development, (201806), vol. 9, doi:10.1016/j.omtm.2018.01.004, XP055675736 | |
| OPPOSITION | - Gabriela Kramer, "In Vitro and in Vivo Comparative Study of Chimeric Liver-Specific Promoters", Molecular Therapy, (200303), vol. 7, doi:10.1016/s1525-0016(02)00060-6, page 3, XP002624075 | |
| OPPOSITION | - Amit C Nathwani, Cecilia Rosales, Jenny Mcintosh, Ghasem Rastegarlari, Devhrut Nathwani, Deepak Raj, Sushmita Nawathe, Simon N Waddington, Roderick Bronson, Scott Jackson, Robert E Donahue, Katherine A High, Federico Mingozzi, Catherine Yc Ng, Junfang Zhou, Yunyu Spence, M Beth Mccarville, Marc Valentine, James Allay, John Coleman, Susan Sleep, John T Gray, Arthur W Nienhuis, Andrew M Davidoff, "Long-term Safety and Efficacy Following Systemic Administration of a Self-complementary AAV Vector Encoding Human FIX Pseudotyped With Serotype 5 and 8 Capsid Proteins", Molecular Therapy, Elsevier Inc., US, US , (20110501), vol. 19, no. 5, doi:10.1038/mt.2010.274, ISSN 1525-0016, pages 876 - 885, XP055405392 | |
| OPPOSITION | - Zy Chen, "Silencing of episomal transgene expression by plasmid bacterial DNA elements in vivo", Gene Therapy, (2004), vol. 11, doi:10.1038/sj.gt.3302231, XP037770458 | |
| OPPOSITION | - F Jacobs, "Direct comparison of hepatocyte-specific expression cassettes following adenoviral and nonviral hydrodynamic gene transfer", Gene Therapy, (2008), vol. 15, doi:10.1038/sj.gt.3303096, XP037778773 | |
| OPPOSITION | - Zhijian Wu, "Optimization of Self-complementary AAV Vectors for Liver-directed Expression Results in Sustained Correction of Hemophilia B at Low Vector Dose", Molecular Therapy, (200802), vol. 16, doi:10.1038/sj.mt.6300355, page 2, XP055097643 | |
| OPPOSITION | - SIMIONI PAOLO ET AL, "X-linked thrombophilia with a mutant factor IX (factor IX Padua).", New England Journal of Medicine, The - NEJM, Massachusetts Medical Society, US, US , (20091022), vol. 361, no. 17, doi:10.1056/NEJMoa0904377, ISSN 1533-4406, pages 1671 - 1675, XP002555909 | |
| OPPOSITION | - Amit C. Nathwani, Edward G.D. Tuddenham, Savita Rangarajan, Cecilia Rosales, Jenny Mcintosh, David C. Linch, Pratima Chowdary, Anne Riddell, Arnulfo Jaquilmac Pie, Chris Harrington, James O'beirne, Keith Smith, John Pasi, Bertil Glader, Pradip Rustagi, Catherine Y.C. Ng, Mark A. Kay, Junfang Zhou, Yunyu Spence, Christopher L. Morton, James Allay, John Coleman, Susan Sleep, John M. Cunningham, Deokumar Srivastava, Etiena Basner-Tschakarjan, Federico Mingozzi, Katherine A. High, John T. Gray, Ulrike M. Reiss, Arthur W. Nienhuis, Andrew M. Davidoff, "Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B", New England Journal of Medicine, New England Journal of Medicine, (20111222), vol. 365, no. 25, doi:10.1056/NEJMoa1108046, ISSN 00284793, pages 2357 - 2365, XP055079598 | |
| OPPOSITION | - Gersappe Anand, Burger Lisa, Pintel David J., "A Premature Termination Codon in Either Exon of Minute Virus of Mice P4 Promoter-generated Pre-mRNA Can Inhibit Nuclear Splicing of the Intervening Intron in an Open Reading Frame-dependent Manner", Journal of Biological Chemistry, American Society for Biochemistry and Molecular Biology, US, US , (19990806), vol. 274, no. 32, doi:10.1074/jbc.274.32.22452, ISSN 0021-9258, pages 22452 - 22458, XP055859671 | |
| OPPOSITION | - Vandendriessche T., Chuah M.K., "Clinical Progress in Gene Therapy: Sustained Partial Correction of the Bleeding Disorder in Patients Suffering from Severe Hemophilia B", HUMAN GENE THERAPY, Mary Ann Liebert, Inc. Publishers, GB, GB , (20120101), vol. 23, no. 1, doi:10.1089/hum.2011.221, ISSN 1043-0342, pages 4 - 6, XP093210527 | |
| OPPOSITION | - Haut Donald D., Pintel D. J., "Intron Definition Is Required for Excision of the Minute Virus of Mice Small Intron and Definition of the Upstream Exon", Journal of Virology, THE AMERICAN SOCIETY FOR MICROBIOLOGY, US, US , (19980301), vol. 72, no. 3, doi:10.1128/JVI.72.3.1834-1843.1998, ISSN 0022-538X, pages 1834 - 1843, XP093210521 | |
| OPPOSITION | - Katherine High, "The gene therapy journey for hemophilia: are we there yet?", Hematology, ASH Education Program, ASH, (20121208), vol. 120, no. 1, doi:10.1182/asheducation-2012.1.375, pages 375 - 381, XP009557383 | |
| OPPOSITION | - NATHWANI AMIT C ET AL, "Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver.", Blood, American Society of Hematology, US, US , (20060401), vol. 107, no. 7, doi:10.1182/blood-2005-10-4035, ISSN 0006-4971, pages 2653 - 2661, XP002534783 | |
| OPPOSITION | - A. C. Nathwani, J. T. Gray, J. Mcintosh, C. Y. C. Ng, J. Zhou, Y. Spence, M. Cochrane, E. Gray, E. G. D. Tuddenham, A. M. Davidoff, "Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates", Blood, American Society of Hematology, US, US , (20070215), vol. 109, no. 4, doi:10.1182/blood-2006-03-010181, ISSN 0006-4971, pages 1414 - 1421, XP055426703 | |
| OPPOSITION | - N. J. Ward, S. M. K. Buckley, S. N. Waddington, T. Vandendriessche, M. K. L. Chuah, A. C. Nathwani, J. Mcintosh, E. G. D. Tuddenham, C. Kinnon, A. J. Thrasher, J. H. Mcvey, "Codon optimization of human factor VIII cDNAs leads to high-level expression", Blood, (20110120), vol. 117, no. 3, doi:10.1182/blood-2010-05-282707, ISSN 00064971, pages 798 - 807, XP055052195 | |
| OPPOSITION | - A. Cantore, N. Nair, P. Della Valle, M. Di Matteo, J. Matrai, F. Sanvito, C. Brombin, C. Di Serio, A. D'angelo, M. Chuah, L. Naldini, T. Vandendriessche, "Hyperfunctional coagulation factor IX improves the efficacy of gene therapy in hemophilic mice", Blood, (20121129), vol. 120, no. 23, doi:10.1182/blood-2012-05-432591, ISSN 00064971, pages 4517 - 4520, XP055050690 | |
| OPPOSITION | - McIntosh J, Lenting P J, Rosales C, Lee D, Rabbanian S, Raj D, Patel N, Tuddenham E G, Christophe O D, McVey J H, Waddington S, Nienhuis A W, Gray J T, Fagone P, Mingozzi F, Zhou S Z, High K A, Cancio M, Ng C Y, Zhou J, Morton C L, Davidoff A M, Nathwani A C, "Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant", Blood, American Society of Hematology, US, US , (20130425), vol. 121, no. 17, doi:10.1182/blood-2012-10-462200, ISSN 0006-4971, pages 3335 - 3344, XP002727999 | |
| OPPOSITION | - Antonio Liras, "Advanced therapies for the treatment of hemophilia: future perspectives", Orphanet Journal of Rare Diseases , (2012), vol. 7, doi:10.1186/1750-1172-7-97, page 97, XP021137537 | |
| OPPOSITION | - Raj Deepak; Davidoff Andrew M; Nathwani Amit C, "Self-complementary adeno-associated viral vectors for gene therapy of hemophilia B: progress and challenges", Expert review of hematology England, Informa UK, UK, UK , (20111101), vol. 4, no. 5, doi:10.1586/ehm.11.48, ISSN 1747-4094, pages 539 - 549, XP009173414 | |
| OPPOSITION | - M. Markusic David, W Herzog Roland, "Liver-Directed Adeno-Associated Viral Gene Therapy for Hemophilia", Journal of Genetic Syndromes & Gene Therapy, OMICS Publishing Group, US, US , (20130403), vol. S1, no. 02, doi:10.4172/2157-7412.S1-009, ISSN 2157-7412, XP093210524 | |
| OTHER | - Anonymous, "Minute virus of mice", Wikipedia, the free encyclopedia, (20210511), Wikipedia, the free encyclopedia, URL: https://en.wikipedia.org/w/index.php?title=Minute_virus_of_mice&oldid=1022543640, (20211110), XP055859669 | - |
| OTHER | - Gersappe Anand, Burger Lisa, Pintel David J., "A Premature Termination Codon in Either Exon of Minute Virus of Mice P4 Promoter-generated Pre-mRNA Can Inhibit Nuclear Splicing of the Intervening Intron in an Open Reading Frame-dependent Manner", Journal of Biological Chemistry, American Society for Biochemistry and Molecular Biology, US, US , (19990806), vol. 274, no. 32, doi:10.1074/jbc.274.32.22452, ISSN 0021-9258, pages 22452 - 22458, XP055859671 | |
| SEARCH | - ZHIJIAN WU ET AL, "Optimization of Self-complementary AAV Vectors for Liver-directed Expression Results in Sustained Correction of Hemophilia B at Low Vector Dose", MOLECULAR THERAPY, (20080201), vol. 16, no. 2, doi:10.1038/sj.mt.6300355, ISSN 1525-0016, pages 280 - 289, XP055097643 [ID] 1,4,8,10,12,14 * the whole document * | |
| SEARCH | - SCHUETTRUMPF JOERG ET AL, "Factor IX variants improve gene therapy efficacy for hemophilia B", BLOOD, AMERICAN SOCIETY OF HEMATOLOGY, US, vol. 105, no. 6, doi:10.1182/BLOOD-2004-08-2990, ISSN 0006-4971, (20050315), pages 2316 - 2323, (20041118), XP002555907 [ID] 1-15 * the whole document * | |
| SEARCH | - A. CANTORE ET AL, "Hyperfunctional coagulation factor IX improves the efficacy of gene therapy in hemophilic mice", BLOOD, (20121004), vol. 120, no. 23, doi:10.1182/blood-2012-05-432591, ISSN 0006-4971, pages 4517 - 4520, XP055050690 [XD] 1-15 * the whole document * | |
