Literature DB >> 35705095

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

Benjamin J Samelson-Jones1.   

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Year:  2022        PMID： 35705095      PMCID： PMC9263299          DOI： 10.1016/j.ymthe.2022.06.002

Source DB:  PubMed          Journal:  Mol Ther        ISSN： 1525-0016            Impact factor:   12.910


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  13 in total

1.  WFH: closing the global gap--achieving optimal care.

Authors:  Mark W Skinner
Journal:  Haemophilia       Date:  2012-07       Impact factor: 4.287

2.  Etranacogene dezaparvovec (AMT-061 phase 2b): normal/near normal FIX activity and bleed cessation in hemophilia B.

Authors:  Annette Von Drygalski; Adam Giermasz; Giancarlo Castaman; Nigel S Key; Susan Lattimore; Frank W G Leebeek; Wolfgang Miesbach; Michael Recht; Alison Long; Robert Gut; Eileen K Sawyer; Steven W Pipe
Journal:  Blood Adv       Date:  2019-11-12

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

Authors:  Robert A French; Benjamin J Samelson-Jones; Glenn P Niemeyer; Clinton D Lothrop; Elizabeth P Merricks; Timothy C Nichols; Valder R Arruda
Journal:  Blood Adv       Date:  2018-03-13

4.  Safety and activity of an engineered, liver-tropic adeno-associated virus vector expressing a hyperactive Padua factor IX administered with prophylactic glucocorticoids in patients with haemophilia B: a single-centre, single-arm, phase 1, pilot trial.

Authors:  Feng Xue; Huiyuan Li; Xia Wu; Wei Liu; Feixu Zhang; Dingyue Tang; Yunfei Chen; Wentian Wang; Ying Chi; Jing Zheng; Zengmin Du; Wei Jiang; Chen Zhong; Jun Wei; Ping Zhu; Rongfeng Fu; Xiaofan Liu; Lingling Chen; Xiaolei Pei; Junjiang Sun; Tao Cheng; Renchi Yang; Xiao Xiao; Lei Zhang
Journal:  Lancet Haematol       Date:  2022-05-19       Impact factor: 30.153

5.  Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant.

Authors:  Lindsey A George; Spencer K Sullivan; Adam Giermasz; John E J Rasko; Benjamin J Samelson-Jones; Jonathan Ducore; Adam Cuker; Lisa M Sullivan; Suvankar Majumdar; Jerome Teitel; Catherine E McGuinn; Margaret V Ragni; Alvin Y Luk; Daniel Hui; J Fraser Wright; Yifeng Chen; Yun Liu; Katie Wachtel; Angela Winters; Stefan Tiefenbacher; Valder R Arruda; Johannes C M van der Loo; Olga Zelenaia; Daniel Takefman; Marcus E Carr; Linda B Couto; Xavier M Anguela; Katherine A High
Journal:  N Engl J Med       Date:  2017-12-07       Impact factor: 91.245

6.  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

7.  X-linked thrombophilia with a mutant factor IX (factor IX Padua).

Authors:  Paolo Simioni; Daniela Tormene; Giulio Tognin; Sabrina Gavasso; Cristiana Bulato; Nicholas P Iacobelli; Jonathan D Finn; Luca Spiezia; Claudia Radu; Valder R Arruda
Journal:  N Engl J Med       Date:  2009-10-22       Impact factor: 91.245

8.  Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response.

Authors:  Catherine S Manno; Glenn F Pierce; Valder R Arruda; Bertil Glader; Margaret Ragni; John J Rasko; John Rasko; Margareth C Ozelo; Keith Hoots; Philip Blatt; Barbara Konkle; Michael Dake; Robin Kaye; Mahmood Razavi; Albert Zajko; James Zehnder; Pradip K Rustagi; Hiroyuki Nakai; Amy Chew; Debra Leonard; J Fraser Wright; Ruth R Lessard; Jürg M Sommer; Michael Tigges; Denise Sabatino; Alvin Luk; Haiyan Jiang; Federico Mingozzi; Linda Couto; Hildegund C Ertl; Katherine A High; Mark A Kay
Journal:  Nat Med       Date:  2006-02-12       Impact factor: 53.440

9.  Factor IX assay discrepancies in the setting of liver gene therapy using a hyperfunctional variant factor IX-Padua.

Authors:  Mary M Robinson; Lindsey A George; Marcus E Carr; Benjamin J Samelson-Jones; Valder R Arruda; John E Murphy; Denis Rybin; Jeremy Rupon; Katherine A High; Stefan Tiefenbacher
Journal:  J Thromb Haemost       Date:  2021-03-28       Impact factor: 5.824

10.  Coagulation factor IX gene transfer to non-human primates using engineered AAV3 capsid and hepatic optimized expression cassette.

Authors:  Sandeep R P Kumar; Jun Xie; Shilang Hu; Jihye Ko; Qifeng Huang; Harrison C Brown; Alok Srivastava; David M Markusic; Christopher B Doering; H Trent Spencer; Arun Srivastava; Guangping Gao; Roland W Herzog
Journal:  Mol Ther Methods Clin Dev       Date:  2021-08-26       Impact factor: 6.698
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