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Literature DB >> 29500218

Complete correction of hemophilia B phenotype by FIX-Padua skeletal muscle gene therapy in an inhibitor-prone dog model.

Robert A French¹, Benjamin J Samelson-Jones¹, Glenn P Niemeyer², Clinton D Lothrop², Elizabeth P Merricks³, Timothy C Nichols³, Valder R Arruda^1,4,5.

Abstract

Entities: Disease Species

Mesh：

Substances：

Year: 2018 PMID： 29500218 PMCID： PMC5851422 DOI： 10.1182/bloodadvances.2017015313

Source DB: PubMed Journal: Blood Adv ISSN： 2473-9529

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25 in total

1. Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B.

Authors: Valder R Arruda; Hansell H Stedman; Virginia Haurigot; George Buchlis; Stefano Baila; Patricia Favaro; Yifeng Chen; Helen G Franck; Shangzhen Zhou; J Fraser Wright; Linda B Couto; Haiyan Jiang; Glenn F Pierce; Dwight A Bellinger; Federico Mingozzi; Timothy C Nichols; Katherine A High
Journal: Blood Date: 2010-03-24 Impact factor: 22.113

2. Impact of AAV Capsid-Specific T-Cell Responses on Design and Outcome of Clinical Gene Transfer Trials with Recombinant Adeno-Associated Viral Vectors: An Evolving Controversy.

Authors: Hildegund C J Ertl; Katherine A High
Journal: Hum Gene Ther Date: 2016-12-29 Impact factor: 5.695

3. Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer.

Authors: Federico Mingozzi; Yi-Lin Liu; Eric Dobrzynski; Antje Kaufhold; Jian Hua Liu; YuQin Wang; Valder R Arruda; Katherine A High; Roland W Herzog
Journal: J Clin Invest Date: 2003-05 Impact factor: 14.808

4. Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model.

Authors: Valder R Arruda; Hansell H Stedman; Timothy C Nichols; Mark E Haskins; Matthew Nicholson; Roland W Herzog; Linda B Couto; Katherine A High
Journal: Blood Date: 2004-10-12 Impact factor: 22.113

5. The efficacy and the risk of immunogenicity of FIX Padua (R338L) in hemophilia B dogs treated by AAV muscle gene therapy.

Authors: Jonathan D Finn; Timothy C Nichols; Nikolaos Svoronos; Elizabeth P Merricks; Dwight A Bellenger; Shangshen Zhou; Paolo Simioni; Katherine A High; Valder R Arruda
Journal: Blood Date: 2012-08-23 Impact factor: 22.113

6. AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B.

Authors: Catherine S Manno; Amy J Chew; Sylvia Hutchison; Peter J Larson; Roland W Herzog; Valder R Arruda; Shing Jen Tai; Margaret V Ragni; Arthur Thompson; Margareth Ozelo; Linda B Couto; Debra G B Leonard; Frederick A Johnson; Alan McClelland; Ciaran Scallan; Erik Skarsgard; Alan W Flake; Mark A Kay; Katherine A High; Bertil Glader
Journal: Blood Date: 2002-12-19 Impact factor: 22.113

7. Gene therapy with adeno-associated virus vector 5-human factor IX in adults with hemophilia B.

Authors: Wolfgang Miesbach; Karina Meijer; Michiel Coppens; Peter Kampmann; Robert Klamroth; Roger Schutgens; Marco Tangelder; Giancarlo Castaman; Joachim Schwäble; Halvard Bonig; Erhard Seifried; Federica Cattaneo; Christian Meyer; Frank W G Leebeek
Journal: Blood Date: 2017-12-15 Impact factor: 22.113

Review 8. Novel approaches to hemophilia therapy: successes and challenges.

Authors: Valder R Arruda; Bhavya S Doshi; Benjamin J Samelson-Jones
Journal: Blood Date: 2017-10-10 Impact factor: 22.113

9. Canine hemophilia B resulting from a point mutation with unusual consequences.

Authors: J P Evans; K M Brinkhous; G D Brayer; H M Reisner; K A High
Journal: Proc Natl Acad Sci U S A Date: 1989-12 Impact factor: 11.205

10. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B.

Authors: 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
Journal: N Engl J Med Date: 2011-12-10 Impact factor: 176.079

14 in total

Review 1. Gene therapy for hemophilia: what does the future hold?

Authors: Bhavya S Doshi; Valder R Arruda
Journal: Ther Adv Hematol Date: 2018-08-27

2. Hyperactivity of factor IX Padua (R338L) depends on factor VIIIa cofactor activity.

Authors: Benjamin J Samelson-Jones; Jonathan D Finn; Lindsey A George; Rodney M Camire; Valder R Arruda
Journal: JCI Insight Date: 2019-06-20

3. Gene Therapy for Inherited Bleeding Disorders.

Authors: Valder R Arruda; Jesse Weber; Benjamin J Samelson-Jones
Journal: Semin Thromb Hemost Date: 2021-02-26 Impact factor: 4.180

4. Worldwide use of factor IX Padua for hemophilia B gene therapy.

Authors: Benjamin J Samelson-Jones
Journal: Mol Ther Date: 2022-06-14 Impact factor: 12.910

Review 5. Role of animal models in biomedical research: a review.

Authors: P Mukherjee; S Roy; D Ghosh; S K Nandi
Journal: Lab Anim Res Date: 2022-07-01

6. Serotype-specific transduction of canine joint tissue explants and cultured monolayers by self-complementary adeno-associated viral vectors.

Authors: Ah Young Kim; Felix Michael Duerr; Jennifer N Phillips; Richard Jude Samulski; Joshua C Grieger; Laurie R Goodrich
Journal: Gene Ther Date: 2022-10-20 Impact factor: 4.184