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Literature DB >> 29274719

Hyperactive Factor IX Padua: A Game-Changer for Hemophilia Gene Therapy.

Thierry VandenDriessche¹, Marinee K Chuah².

Abstract

Entities: Disease

Mesh：

Substances：
factor IX-Padua
Factor IX

Year: 2017 PMID： 29274719 PMCID： PMC5763158 DOI： 10.1016/j.ymthe.2017.12.007

Source DB: PubMed Journal: Mol Ther ISSN： 1525-0016 Impact factor: 11.454

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17 in total

1. High plasma concentration of factor VIIIc is a major risk factor for venous thromboembolism.

Authors: R A Kraaijenhagen; P S in't Anker; M M Koopman; P H Reitsma; M H Prins; A van den Ende; H R Büller
Journal: Thromb Haemost Date: 2000-01 Impact factor: 5.249

2. Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector.

Authors: M A Kay; C S Manno; M V Ragni; P J Larson; L B Couto; A McClelland; B Glader; A J Chew; S J Tai; R W Herzog; V Arruda; F Johnson; C Scallan; E Skarsgard; A W Flake; K A High
Journal: Nat Genet Date: 2000-03 Impact factor: 38.330

Review 3. Cellular and genetic therapies for haemophilia.

Authors: D Lillicrap; T VandenDriessche; K High
Journal: Haemophilia Date: 2006-07 Impact factor: 4.287

4. AAV5-Factor VIII Gene Transfer in Severe Hemophilia A.

Authors: Savita Rangarajan; Liron Walsh; Will Lester; David Perry; Bella Madan; Michael Laffan; Hua Yu; Christian Vettermann; Glenn F Pierce; Wing Y Wong; K John Pasi
Journal: N Engl J Med Date: 2017-12-09 Impact factor: 91.245

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. Liver-directed lentiviral gene therapy in a dog model of hemophilia B.

Authors: Alessio Cantore; Marco Ranzani; Cynthia C Bartholomae; Monica Volpin; Patrizia Della Valle; Francesca Sanvito; Lucia Sergi Sergi; Pierangela Gallina; Fabrizio Benedicenti; Dwight Bellinger; Robin Raymer; Elizabeth Merricks; Francesca Bellintani; Samia Martin; Claudio Doglioni; Armando D'Angelo; Thierry VandenDriessche; Marinee K Chuah; Manfred Schmidt; Timothy Nichols; Eugenio Montini; Luigi Naldini
Journal: Sci Transl Med Date: 2015-03-04 Impact factor: 17.956

8. Long-term safety and efficacy of factor IX gene therapy in hemophilia B.

Authors: Amit C Nathwani; Ulreke M Reiss; Edward G D Tuddenham; Cecilia Rosales; Pratima Chowdary; Jenny McIntosh; Marco Della Peruta; Elsa Lheriteau; Nishal Patel; Deepak Raj; Anne Riddell; Jun Pie; Savita Rangarajan; David Bevan; Michael Recht; Yu-Min Shen; Kathleen G Halka; Etiena Basner-Tschakarjan; Federico Mingozzi; Katherine A High; James Allay; Mark A Kay; Catherine Y C Ng; Junfang Zhou; Maria Cancio; Christopher L Morton; John T Gray; Deokumar Srivastava; Arthur W Nienhuis; Andrew M Davidoff
Journal: N Engl J Med Date: 2014-11-20 Impact factor: 176.079

9. Employing a gain-of-function factor IX variant R338L to advance the efficacy and safety of hemophilia B human gene therapy: preclinical evaluation supporting an ongoing adeno-associated virus clinical trial.

Authors: Paul E Monahan; Junjiang Sun; Tong Gui; Genlin Hu; William B Hannah; David G Wichlan; Zhijian Wu; Joshua C Grieger; Chengwen Li; Thipparat Suwanmanee; Darrel W Stafford; Carmen J Booth; Jade J Samulski; Tal Kafri; Scott W J McPhee; R Jude Samulski
Journal: Hum Gene Ther Date: 2015-01-21 Impact factor: 5.695

Hyperactive Factor IX Padua: A Game-Changer for Hemophilia Gene Therapy.

1. High plasma concentration of factor VIIIc is a major risk factor for venous thromboembolism.

2. Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector.

Review 3. Cellular and genetic therapies for haemophilia.

4. AAV5-Factor VIII Gene Transfer in Severe Hemophilia A.

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

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

7. Liver-directed lentiviral gene therapy in a dog model of hemophilia B.

8. Long-term safety and efficacy of factor IX gene therapy in hemophilia B.

9. Employing a gain-of-function factor IX variant R338L to advance the efficacy and safety of hemophilia B human gene therapy: preclinical evaluation supporting an ongoing adeno-associated virus clinical trial.

10. Hyperactive piggyBac transposons for sustained and robust liver-targeted gene therapy.

1. Evolutionary insights into coagulation factor IX Padua and other high-specific-activity variants.

2. Gene Therapy Advances: A Meta-Analysis of AAV Usage in Clinical Settings.

Review 3. Immunogenicity and toxicity of AAV gene therapy.

Review 4. Protein-Engineered Coagulation Factors for Hemophilia Gene Therapy.