Artículos de revistas sobre el tema "AAV-FIX"
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Bharadwaj, Arpita S., Meagan Kelly, Dongsoo Kim y Hengjun Chao. "Induction of immune tolerance to FIX by intramuscular AAV gene transfer is independent of the activation status of dendritic cells". Blood 115, n.º 3 (21 de enero de 2010): 500–509. http://dx.doi.org/10.1182/blood-2009-08-239509.
Texto completoArruda, Valder R., Hansell H. Stedman, Virginia Haurigot, George Buchlis, Stefano Baila, Patricia Favaro, Yifeng Chen et al. "Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B". Blood 115, n.º 23 (10 de junio de 2010): 4678–88. http://dx.doi.org/10.1182/blood-2009-12-261156.
Texto completoSchuettrumpf, Joerg, Jianxiang Zou, Shin Jen Tai, Alexander Schlachterman, Kian Tian, Shyrie Edmonson, Jianhua Liu, Patricia Andrade-Gordon, Katherine High y Valder Arruda. "A Novel Role of Coagulation Proteases on Viral-Based Gene Transfer Efficacy." Blood 104, n.º 11 (16 de noviembre de 2004): 691. http://dx.doi.org/10.1182/blood.v104.11.691.691.
Texto completoJiang, Haiyan, Linda B. Couto, Susannah Patarroyo-White, Tongyao Liu, Dea Nagy, Joseph A. Vargas, Shangzhen Zhou et al. "Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy". Blood 108, n.º 10 (15 de noviembre de 2006): 3321–28. http://dx.doi.org/10.1182/blood-2006-04-017913.
Texto completoKao, Chung-Yang, Chia-Ni Lin, I.-Shing Yu, Mi-Hua Tao, Hua-Lin Wu, Guey-Yueh Shi, Yung-Li Yang, Jau-Tsuen Kao y Shu-Wha Lin. "FIX-Triple, a gain-of-function factor IX variant, improves haemostasis in mouse models without increased risk of thrombosis". Thrombosis and Haemostasis 104, n.º 08 (2010): 355–65. http://dx.doi.org/10.1160/th09-11-0792.
Texto completoKao, Chung-Yang, Shu-Jhu Yang, Mi-Hua Tao, Yung-Ming Jeng, I.-Shing Yu y Shu-Wha Lin. "Incorporation of the factor IX Padua mutation into FIX-Triple improves clotting activity in vitro and in vivo". Thrombosis and Haemostasis 110, n.º 08 (2013): 244–56. http://dx.doi.org/10.1160/th13-02-0154.
Texto completoWang, Lili, Yang Yang, Camilo Ayala Breton, John White, Jia Zhang, Yan Che, Alexei Saveliev et al. "CRISPR/Cas9-mediated in vivo gene targeting corrects hemostasis in newborn and adult factor IX–knockout mice". Blood 133, n.º 26 (27 de junio de 2019): 2745–52. http://dx.doi.org/10.1182/blood.2019000790.
Texto completoBaila, Stefano, Christian Furlan Freguia, Nicholas Iacobelli, Danielle Dunn, Joerg Schuettrumpf, Federico Mingozzi, Patricia Andrade-Gordon y Valder R. Arruda. "Protease−Activated Receptor 2 (PAR−2) as a Novel Target To Prevent Inhibitor Formation to FIX." Blood 108, n.º 11 (16 de noviembre de 2006): 763. http://dx.doi.org/10.1182/blood.v108.11.763.763.
Texto completoKelly, Meagan E., Jiacai Zhuo, Arpita S. Bharadwaj y Hengjun Chao. "Induction of Immune Tolerance to FIX Following Muscular AAV Gene Transfer Is AAV-dose/FIX-level Dependent". Molecular Therapy 17, n.º 5 (mayo de 2009): 857–63. http://dx.doi.org/10.1038/mt.2009.25.
Texto completoMarkusic, David M. y Brett Palaschak. "Defining the Risk of Suboptimal AAV8-F9 Vector Delivery in Inhibitor Positive Hemophilia B Mice". Blood 128, n.º 22 (2 de diciembre de 2016): 3508. http://dx.doi.org/10.1182/blood.v128.22.3508.3508.
Texto completoFinn, Jonathan D., Timothy C. Nichols, Nikolaos Svoronos, Elizabeth P. Merricks, Dwight A. Bellenger, Shangshen Zhou, Paolo Simioni, Katherine A. High y Valder R. Arruda. "The efficacy and the risk of immunogenicity of FIX Padua (R338L) in hemophilia B dogs treated by AAV muscle gene therapy". Blood 120, n.º 23 (29 de noviembre de 2012): 4521–23. http://dx.doi.org/10.1182/blood-2012-06-440123.
Texto completoFrench, Robert, Nicholas Martin, Timothy C. Nichols, Glenn P. Niemeyer, Clinton D. Lothrop y Valder R. Arruda. "Complete Correction of Severe Canine Hemophilia B By Skeletal Muscle Directed AAV-Based FIX-Padua Gene Therapy in Inhibitor-Prone Dogs". Blood 126, n.º 23 (3 de diciembre de 2015): 3487. http://dx.doi.org/10.1182/blood.v126.23.3487.3487.
Texto completoHigh, Katherine, Michael Tigges, Catherine Manno, Denise Sabatino, Valder Arruda, Roland Herzog, Pradip Rustagi et al. "Human Immune Responses to AAV-2 Capsid May Limit Duration of Expression in Liver-Directed Gene Transfer in Humans with Hemophilia B." Blood 104, n.º 11 (16 de noviembre de 2004): 413. http://dx.doi.org/10.1182/blood.v104.11.413.413.
Texto completoBaila, Stefano, Christian Furlan Freguia, Daniel Orr, Federico Mingozzi, Joerge Schuettrumpf y Valder R. Arruda. "Protease-Activated Receptor-2 (PAR-2) as a Novel Target for Modulating Immune Responses to Neo Antigens Following In Vivo Gene Transfer." Blood 106, n.º 11 (16 de noviembre de 2005): 1296. http://dx.doi.org/10.1182/blood.v106.11.1296.1296.
Texto completoGe, Ying, Sandra Powell, Melinda Van Roey y James G. McArthur. "Factors influencing the development of an anti–factor IX (FIX) immune response following administration of adeno-associated virus–FIX". Blood 97, n.º 12 (15 de junio de 2001): 3733–37. http://dx.doi.org/10.1182/blood.v97.12.3733.
Texto completoScallan, Ciaran D., Haiyan Jiang, Tongyao Liu, Susannah Patarroyo-White, Jurg M. Sommer, Shangzhen Zhou, Linda B. Couto y Glenn F. Pierce. "Human immunoglobulin inhibits liver transduction by AAV vectors at low AAV2 neutralizing titers in SCID mice". Blood 107, n.º 5 (1 de marzo de 2006): 1810–17. http://dx.doi.org/10.1182/blood-2005-08-3229.
Texto completoNathwani, Amit C., Edward GD Tuddenham, Savita Rangarajan, Cecilia Rosales, Jenny H. McIntosh, David C. Linch, Pratima Chowdary et al. "Adeno-Associated Viral Vector Mediated Gene Transfer for Hemophilia B". Blood 118, n.º 21 (18 de noviembre de 2011): 5. http://dx.doi.org/10.1182/blood.v118.21.5.5.
Texto completoNathwani, Amit C. "Gene therapy for hemophilia". Hematology 2019, n.º 1 (6 de diciembre de 2019): 1–8. http://dx.doi.org/10.1182/hematology.2019000007.
Texto completoSamelson-Jones, Benjamin J., John D. Finn, Rodney M. Camire y Valder R. Arruda. "The Complete Dependence of Factor IX Padua (R338L) Hyperactivity on Factor VIIIa Cofactor Activity Supports Its Safety As a Transgene for Hemophilia B Gene Therapy". Blood 132, Supplement 1 (29 de noviembre de 2018): 3486. http://dx.doi.org/10.1182/blood-2018-99-119270.
Texto completoMount, Jane D., Roland W. Herzog, D. Michael Tillson, Susan A. Goodman, Nancy Robinson, Mark L. McCleland, Dwight Bellinger et al. "Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy". Blood 99, n.º 8 (15 de abril de 2002): 2670–76. http://dx.doi.org/10.1182/blood.v99.8.2670.
Texto completoMonahan, Paul E., Junjiang Sun, Tong Gui, David G. Wichlan, Scott W. McPhee y R. Jude Samulski. "Employing Factor IX Variants to Avoid Limitations Imposed by Immune Recognition of AAV Vector in Hemophilia B Gene Therapy". Blood 118, n.º 21 (18 de noviembre de 2011): 3124. http://dx.doi.org/10.1182/blood.v118.21.3124.3124.
Texto completoVerhoef, Daniel, Jonathan H. Foley, Andrew Goodale, Emma Macrae, Jenny McIntosh, Romuald Corbau, Keith Gomez, Anne Riddell y Amit C. Nathwani. "A Novel Lysine to Arginine Substitution at Position 301 Enhances Activity of Factor IX". Blood 132, Supplement 1 (29 de noviembre de 2018): 3772. http://dx.doi.org/10.1182/blood-2018-99-112832.
Texto completoChao, Hengjun, Wei Chen, James R. Tunstead y Christopher E. Walsh. "Human Factor IX Expression and Hemophilia Phenotypic Correction in Mice: A Comparison of AAV Serotypes." Blood 104, n.º 11 (16 de noviembre de 2004): 3985. http://dx.doi.org/10.1182/blood.v104.11.3985.3985.
Texto completoCao, Ou, Eric Dobrzynski, Lixin Wang, Sushrusha Nayak, Bethany Mingle, Cox Terhorst y Roland W. Herzog. "Induction and role of regulatory CD4+CD25+ T cells in tolerance to the transgene product following hepatic in vivo gene transfer". Blood 110, n.º 4 (15 de agosto de 2007): 1132–40. http://dx.doi.org/10.1182/blood-2007-02-073304.
Texto completoNiemeyer, Glenn P., Roland W. Herzog, Jane Mount, Valder R. Arruda, D. Michael Tillson, John Hathcock, Frederik W. van Ginkel, Katherine A. High y Clinton D. Lothrop. "Long-term correction of inhibitor-prone hemophilia B dogs treated with liver-directed AAV2-mediated factor IX gene therapy". Blood 113, n.º 4 (22 de enero de 2009): 797–806. http://dx.doi.org/10.1182/blood-2008-10-181479.
Texto completoNiemeyer, Glenn P., Roland W. Herzog, Jane D. Mount, Valder Arruda, Michael Tillson, John Hathcock, Katherine A. High y Clinton D. Lothrop. "Six-Year Follow-Up of Inhibitor Prone Hemophilia B Dogs Treated with Muscle and Liver-Directed AAV2 Mediated Factor IX Gene Therapy." Blood 108, n.º 11 (16 de noviembre de 2006): 3282. http://dx.doi.org/10.1182/blood.v108.11.3282.3282.
Texto completoSchroeder, Jocelyn A., Juan Chen, Yingyu Chen, Yuanhua Cai, Hongyin Yu, Jeremy G. Mattson, Paul E. Monahan y Qizhen Shi. "Platelet-targeted hyperfunctional FIX gene therapy for hemophilia B mice even with preexisting anti-FIX immunity". Blood Advances 5, n.º 5 (1 de marzo de 2021): 1224–38. http://dx.doi.org/10.1182/bloodadvances.2020004071.
Texto completoSchlachterman, Alexander, Jianhua Liu, Majed Aljamali, Valder R. Arruda y Katherine A. High. "Assessing Hemostatic Efficacy of Continuous Expression of Factor VIIa Following Administration of AAV Vectors into Hemophilia B Mice." Blood 104, n.º 11 (16 de noviembre de 2004): 3184. http://dx.doi.org/10.1182/blood.v104.11.3184.3184.
Texto completoHigh, Katherine A. "The gene therapy journey for hemophilia: are we there yet?" Blood 120, n.º 23 (29 de noviembre de 2012): 4482–87. http://dx.doi.org/10.1182/blood-2012-05-423210.
Texto completoCohn, Ellen F., Meagan E. Kelly, Jiacai Zhuo y Hengjun Chao. "Efficient Induction of Tolerance to FIX by Direct Intramuscular Delivery of AAV1." Blood 108, n.º 11 (16 de noviembre de 2006): 3287. http://dx.doi.org/10.1182/blood.v108.11.3287.3287.
Texto completoCrudele, Julie M., Jonathan D. Finn, Nicholas B. Martin, Joshua I. Siner, Yifeng Chen, Shangzhen Zhou, Glenn Niemeyer et al. "Tolerance Induction To FIX Padua With AAV Liver Gene Transfer In Inhibitor-Prone Hemophilia B Dogs". Blood 122, n.º 21 (15 de noviembre de 2013): 4203. http://dx.doi.org/10.1182/blood.v122.21.4203.4203.
Texto completoHigh, Katherine A. "The gene therapy journey for hemophilia: are we there yet?" Hematology 2012, n.º 1 (8 de diciembre de 2012): 375–81. http://dx.doi.org/10.1182/asheducation.v2012.1.375.3797885.
Texto completoDavidoff, Andrew, Edward GD Tuddenham, Savita Rangarajan, Cecilia Rosales, Jenny McIntosh, Pratima Chowdary, Anne Riddell et al. "Stable Factor IX Activity Following AAV-Mediated Gene Transfer in Patients with Severe Hemophilia B". Blood 120, n.º 21 (16 de noviembre de 2012): 752. http://dx.doi.org/10.1182/blood.v120.21.752.752.
Texto completoHui, Daniel J., Etiena Basner-Tschakarjan, Gary C. Pien, William D. Martin, Annie S. DeGroot, Katherine A. High y Federico Mingozzi. "Peptide-Induced Antigen-Specific CD4+CD25+FoxP3+ T Cells Suppress Cytotoxicity T Cell Responses Directed Against the AAV Capsid." Blood 116, n.º 21 (19 de noviembre de 2010): 3769. http://dx.doi.org/10.1182/blood.v116.21.3769.3769.
Texto completoLi, Hojun, Nirav Malani, Shari R. Hamilton, Alexander Schlachterman, Jim Z. Zhang, Giulio Bussadori, Shyrie A. Edmonson et al. "Assessment of Insertional Mutagenesis Risk Following AAV Vector-Mediated Factor IX Gene Transfer in Mice." Blood 114, n.º 22 (20 de noviembre de 2009): 2465. http://dx.doi.org/10.1182/blood.v114.22.2465.2465.
Texto completoCrudele, Julie M., Jonathan D. Finn, Joshua I. Siner, Nicholas B. Martin, Glenn P. Niemeyer, Shangzhen Zhou, Federico Mingozzi, Clinton D. Lothrop y Valder R. Arruda. "AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice". Blood 125, n.º 10 (5 de marzo de 2015): 1553–61. http://dx.doi.org/10.1182/blood-2014-07-588194.
Texto completoNguyen, Giang N., John K. Everett, Hayley Raymond, Samita Kafle, Elizabeth P. Merricks, Haig H. Kazazian, Timothy C. Nichols, Frederic D. Bushman y Denise E. Sabatino. "Long-Term AAV-Mediated Factor VIII Expression in Nine Hemophilia A Dogs: A 10 Year Follow-up Analysis on Durability, Safety and Vector Integration". Blood 134, Supplement_1 (13 de noviembre de 2019): 611. http://dx.doi.org/10.1182/blood-2019-126007.
Texto completoDobrzynski, E., F. Mingozzi, L. Wang, B. Mingle, O. Cao y R. W. Herzog. "Hepatic Tolerance Induction Prevents Inflammatory Responses To Factor IX Expressing Cells at Supplementary Sites of Gene Transfer." Blood 104, n.º 11 (16 de noviembre de 2004): 3185. http://dx.doi.org/10.1182/blood.v104.11.3185.3185.
Texto completoFinn, Jonathan D., Daniel J. Hui, Downey Harre, Danielle Dunn, Federico Mingozzi, Shangzhen Zhou y Katherine A. High. "Proteasome Inhibitors Decrease AAV2 Capsid-Derived Peptide Epitope Presentation On MHC Class I Following Transduction." Blood 114, n.º 22 (20 de noviembre de 2009): 695. http://dx.doi.org/10.1182/blood.v114.22.695.695.
Texto completoSun, Junjiang, Narine Hakobyan, Leonard A. Valentino, Brian L. Feldman, R. Jude Samulski y Paul E. Monahan. "Intraarticular factor IX protein or gene replacement protects against development of hemophilic synovitis in the absence of circulating factor IX". Blood 112, n.º 12 (1 de diciembre de 2008): 4532–41. http://dx.doi.org/10.1182/blood-2008-01-131417.
Texto completoWeiller, Markus, Helen Wang, Sogué Coulibaly, Maria Schuster, Hanspeter Rottensteiner, Kefeng Sun, Marinee K. Chuah, Thierry Vandendriessche, Friedrich Scheiflinger y Werner Höllriegl. "Evaluation of the Human Factor IX Gene Therapy Vector TAK-748 in Hemophilia: Results from Non-Clinical Studies in Factor IX Knockout Mice and Rhesus Monkeys". Blood 134, Supplement_1 (13 de noviembre de 2019): 4633. http://dx.doi.org/10.1182/blood-2019-124734.
Texto completoGiangrande, Paul. "The Future of Hemophilia Treatment: Longer-Acting Factor Concentrates versus Gene Therapy". Seminars in Thrombosis and Hemostasis 42, n.º 05 (5 de mayo de 2016): 513–17. http://dx.doi.org/10.1055/s-0036-1579637.
Texto completoNathwani, Amit C., John T. Gray, Catherine Y. C. Ng, Junfang Zhou, Yunyu Spence, Simon N. Waddington, Edward G. D. Tuddenham 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 107, n.º 7 (1 de abril de 2006): 2653–61. http://dx.doi.org/10.1182/blood-2005-10-4035.
Texto completoFinn, Jonathan D., Paolo Simioni, Nicholas Iacobelli, Shangzhen Zhou, Timothy C. Nichols, Katherine A. High y Valder R. Arruda. "FIX-R338L (FIX Padua) as a Successful Alternative for the Treatment of Canine Severe Hemophilia B." Blood 114, n.º 22 (20 de noviembre de 2009): 694. http://dx.doi.org/10.1182/blood.v114.22.694.694.
Texto completoWang, Xiaomei, Alexandra Sherman, Jin Su, Henry Daniell y Roland W. Herzog. "Mechanism Of Oral Tolerance Induced By Bioencapsulated Coagulation Factor IX In Hemophilia B Mice". Blood 122, n.º 21 (15 de noviembre de 2013): 30. http://dx.doi.org/10.1182/blood.v122.21.30.30.
Texto completoLillicrap, David. "Genetic Characterization of Hemophilia and Implications for Novel Therapies". Blood 116, n.º 21 (19 de noviembre de 2010): SCI—9—SCI—9. http://dx.doi.org/10.1182/blood.v116.21.sci-9.sci-9.
Texto completoDane, Allison, Jenny McIntosh, Doyoung Lee, Rose Sheridan, Mark Maginn, Romuald Corbau, Andrew M. Davidoff y Amit C. Nathwani. "Preclinical Evaluation of an Engineered AAV Capsid in Non-Human Primates for the Treatment of Haemophilia B". Blood 132, Supplement 1 (29 de noviembre de 2018): 2197. http://dx.doi.org/10.1182/blood-2018-99-117969.
Texto completoChen, Yingyu, Jocelyn A. Schroeder, Erin L. Kuether, Guowei Zhang, Robert R. Montgomery y Qizhen Shi. "Lentivirus-Mediated Platelet Gene Therapy Corrects Bleeding Diathesis and Induces Immune Tolerance in Murine Hemophilia B Mice". Blood 120, n.º 21 (16 de noviembre de 2012): 1101. http://dx.doi.org/10.1182/blood.v120.21.1101.1101.
Texto completoArruda, Valder R. "Hemophilia Gene Therapy". Blood 118, n.º 21 (18 de noviembre de 2011): SCI—48—SCI—48. http://dx.doi.org/10.1182/blood.v118.21.sci-48.sci-48.
Texto completoLin, Chia-Ni, Chung-Yang Kao, Chia-Lun Hong, Peiqing Ye, Carol H. Miao, Nobuko Hamaguchi, Hua-Lin Wu et al. "Engineered Factor IX with Augmented Clotting Activities in a Hemophilia B Mouse Model." Blood 112, n.º 11 (16 de noviembre de 2008): 2025. http://dx.doi.org/10.1182/blood.v112.11.2025.2025.
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