Literature DB >> 23281440

Gene therapy for hemophilia: addressing the coming challenges of affordability and accessibility.

Mark W Skinner.   

Abstract

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Year:  2013        PMID： 23281440      PMCID： PMC3538323          DOI： 10.1038/mt.2012.272

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


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  5 in total

1.  Cell phones and landlines: the impact of gene therapy on the cost and availability of treatment for hemophilia.

Authors:  Katherine A High; Mark W Skinner
Journal:  Mol Ther       Date:  2011-10       Impact factor: 11.454

2.  Hemophilia gene therapy: a Holy Grail found.

Authors:  Katherine P Ponder
Journal:  Mol Ther       Date:  2011-03       Impact factor: 11.454

Review 3.  Treatment of haemophilia A and B and von Willebrand's disease: summary and conclusions of a systematic review as part of a Swedish health-technology assessment.

Authors:  E Berntorp; J Astermark; F Baghaei; D Bergqvist; M Holmström; B Ljungberg; A Norlund; J Palmblad; P Petrini; L Stigendal; J Säwe
Journal:  Haemophilia       Date:  2011-12-12       Impact factor: 4.287

4.  Comparing prophylaxis with episodic treatment in haemophilia A: implications for clinical practice.

Authors:  M Manco-Johnson
Journal:  Haemophilia       Date:  2007-09       Impact factor: 4.287

5.  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
  5 in total
  7 in total

1.  A Low Protein Binding Cationic Poly(2-oxazoline) as Non-Viral Vector.

Authors:  Zhijian He; Lei Miao; Rainer Jordan; Devika S-Manickam; Robert Luxenhofer; Alexander V Kabanov
Journal:  Macromol Biosci       Date:  2015-04-02       Impact factor: 4.979

Review 2.  Hemophilia gene therapy comes of age.

Authors:  Lindsey A George
Journal:  Blood Adv       Date:  2017-12-08

Review 3.  Recent advances in hemophilia B therapy.

Authors:  Sarena D Horava; Nicholas A Peppas
Journal:  Drug Deliv Transl Res       Date:  2017-06       Impact factor: 4.617

Review 4.  Hemophilia gene therapy comes of age.

Authors:  Lindsey A George
Journal:  Hematology Am Soc Hematol Educ Program       Date:  2017-12-08

Review 5.  The role of patient and healthcare professionals in the era of new hemophilia treatments in developed and developing countries.

Authors:  Fadi Nossair; Courtney D Thornburg
Journal:  Ther Adv Hematol       Date:  2018-07-02

Review 6.  Translational data from adeno-associated virus-mediated gene therapy of hemophilia B in dogs.

Authors:  Timothy C Nichols; Margaret H Whitford; Valder R Arruda; Hansell H Stedman; Mark A Kay; Katherine A High
Journal:  Hum Gene Ther Clin Dev       Date:  2015-02-12

7.  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
  7 in total

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