Literature DB >> 25397928

Gene therapy for hemophilia: the clot thickens.

Katherine A High1.   

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Year:  2014        PMID： 25397928      PMCID： PMC4236063          DOI： 10.1089/hum.2014.2541

Source DB:  PubMed          Journal:  Hum Gene Ther        ISSN： 1043-0342            Impact factor:   5.695


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  45 in total

Review 1.  Establishing haemophilia care in developing countries: using data to overcome the barrier of pessimism.

Authors:  B L Evatt; L Robillard
Journal:  Haemophilia       Date:  2000-05       Impact factor: 4.287

2.  Effects of HIV infection on age and cause of death for persons with hemophilia A in the United States.

Authors:  T L Chorba; R C Holman; M J Clarke; B L Evatt
Journal:  Am J Hematol       Date:  2001-04       Impact factor: 10.047

3.  Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer.

Authors:  George Buchlis; Gregory M Podsakoff; Antonetta Radu; Sarah M Hawk; Alan W Flake; Federico Mingozzi; Katherine A High
Journal:  Blood       Date:  2012-01-23       Impact factor: 22.113

4.  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

5.  Lack of germline transmission of vector sequences following systemic administration of recombinant AAV-2 vector in males.

Authors:  V R Arruda; P A Fields; R Milner; L Wainwright; M P De Miguel; P J Donovan; R W Herzog; T C Nichols; J A Biegel; M Razavi; M Dake; D Huff; A W Flake; L Couto; M A Kay; K A High
Journal:  Mol Ther       Date:  2001-12       Impact factor: 11.454

6.  Nonviral transfer of the gene encoding coagulation factor VIII in patients with severe hemophilia A.

Authors:  D A Roth; N E Tawa; J M O'Brien; D A Treco; R F Selden
Journal:  N Engl J Med       Date:  2001-06-07       Impact factor: 91.245

7.  Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy.

Authors:  Jane D Mount; Roland W Herzog; D Michael Tillson; Susan A Goodman; Nancy Robinson; Mark L McCleland; Dwight Bellinger; Timothy C Nichols; Valder R Arruda; Clinton D Lothrop; Katherine A High
Journal:  Blood       Date:  2002-04-15       Impact factor: 22.113

8.  The Chapel Hill hemophilia A dog colony exhibits a factor VIII gene inversion.

Authors:  Jay N Lozier; Amalia Dutra; Evgenia Pak; Nan Zhou; Zhili Zheng; Timothy C Nichols; Dwight A Bellinger; Marjorie Read; Richard A Morgan
Journal:  Proc Natl Acad Sci U S A       Date:  2002-09-19       Impact factor: 11.205

Review 9.  Immune responses to AAV vectors: overcoming barriers to successful gene therapy.

Authors:  Federico Mingozzi; Katherine A High
Journal:  Blood       Date:  2013-04-17       Impact factor: 22.113

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
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  7 in total

1.  Dexamethasone Transiently Enhances Transgene Expression in the Liver When Administered at Late-Phase Post Long-Term Adeno-Associated Virus Transduction.

Authors:  Zheng Chai; Xintao Zhang; Amanda Lee Dobbins; Richard Jude Samulski; Elizabeth P Merricks; Timothy C Nichols; Chengwen Li
Journal:  Hum Gene Ther       Date:  2022-01-06       Impact factor: 4.793

2.  Clinical and imaging analysis of subclinical hemophilia combined with coxarthrosis: case report and literature review.

Authors:  Cheng Liu; Jun Guo; Qiu Cui; Dingfeng Li; Yanjun Zeng
Journal:  Springerplus       Date:  2016-12-01

3.  Syngeneic AAV Pseudo-particles Potentiate Gene Transduction of AAV Vectors.

Authors:  Qizhao Wang; Biao Dong; Katie A Pokiniewski; Jenni Firrman; Zhongren Wu; Mario P S Chin; Xiongwen Chen; LinShu Liu; Ruian Xu; Yong Diao; Weidong Xiao
Journal:  Mol Ther Methods Clin Dev       Date:  2016-12-24       Impact factor: 6.698

4.  Defining the Optimal FVIII Transgene for Placental Cell-Based Gene Therapy to Treat Hemophilia A.

Authors:  Nadia El-Akabawy; Martin Rodriguez; Ritu Ramamurthy; Andrew Rabah; Brady Trevisan; Alshaimaa Morsi; Sunil George; Jordan Shields; Diane Meares; Andrew Farland; Anthony Atala; Christopher B Doering; H Trent Spencer; Christopher D Porada; Graça Almeida-Porada
Journal:  Mol Ther Methods Clin Dev       Date:  2020-03-14       Impact factor: 6.698

Review 5.  Organoids and microphysiological systems: Promising models for accelerating AAV gene therapy studies.

Authors:  Ritu Mahesh Ramamurthy; Anthony Atala; Christopher D Porada; Graҫa Almeida-Porada
Journal:  Front Immunol       Date:  2022-09-26       Impact factor: 8.786

Review 6.  Clinical Considerations for Capsid Choice in the Development of Liver-Targeted AAV-Based Gene Transfer.

Authors:  Steven Pipe; Frank W G Leebeek; Valerie Ferreira; Eileen K Sawyer; John Pasi
Journal:  Mol Ther Methods Clin Dev       Date:  2019-09-10       Impact factor: 6.698

7.  CRISPR/Cas9-mediated knockin of human factor IX into swine factor IX locus effectively alleviates bleeding in hemophilia B pigs.

Authors:  Jiahuan Chen; Beiying An; Biao Yu; Xiaohuan Peng; Hongming Yuan; Qiangbing Yang; Xue Chen; Tingting Yu; Lingyu Wang; Xinwei Zhang; He Wang; Xiaodong Zou; Daxin Pang; Hongsheng Ouyang; Xiaochun Tang
Journal:  Haematologica       Date:  2021-03-01       Impact factor: 9.941
  7 in total

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