Literature DB >> 25943496

Moving forward toward a cure for hemophilia B.

Thierry VandenDriessche1, Marinee K Chuah2.   

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Year:  2015        PMID: 25943496      PMCID: PMC4427887          DOI: 10.1038/mt.2015.56

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


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  25 in total

1.  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

2.  AAV-1-mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells.

Authors:  Federico Mingozzi; Janneke J Meulenberg; Daniel J Hui; Etiena Basner-Tschakarjan; Nicole C Hasbrouck; Shyrie A Edmonson; Natalie A Hutnick; Michael R Betts; John J Kastelein; Erik S Stroes; Katherine A High
Journal:  Blood       Date:  2009-06-08       Impact factor: 22.113

3.  Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus.

Authors:  R W Herzog; J N Hagstrom; S H Kung; S J Tai; J M Wilson; K J Fisher; K A High
Journal:  Proc Natl Acad Sci U S A       Date:  1997-05-27       Impact factor: 11.205

4.  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

5.  Effect of gene therapy on visual function in Leber's congenital amaurosis.

Authors:  James W B Bainbridge; Alexander J Smith; Susie S Barker; Scott Robbie; Robert Henderson; Kamaljit Balaggan; Ananth Viswanathan; Graham E Holder; Andrew Stockman; Nick Tyler; Simon Petersen-Jones; Shomi S Bhattacharya; Adrian J Thrasher; Fred W Fitzke; Barrie J Carter; Gary S Rubin; Anthony T Moore; Robin R Ali
Journal:  N Engl J Med       Date:  2008-04-27       Impact factor: 91.245

6.  Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial.

Authors:  Albert M Maguire; Katherine A High; Alberto Auricchio; J Fraser Wright; Eric A Pierce; Francesco Testa; Federico Mingozzi; Jeannette L Bennicelli; Gui-shuang Ying; Settimio Rossi; Ann Fulton; Kathleen A Marshall; Sandro Banfi; Daniel C Chung; Jessica I W Morgan; Bernd Hauck; Olga Zelenaia; Xiaosong Zhu; Leslie Raffini; Frauke Coppieters; Elfride De Baere; Kenneth S Shindler; Nicholas J Volpe; Enrico M Surace; Carmela Acerra; Arkady Lyubarsky; T Michael Redmond; Edwin Stone; Junwei Sun; Jennifer Wellman McDonnell; Bart P Leroy; Francesca Simonelli; Jean Bennett
Journal:  Lancet       Date:  2009-10-23       Impact factor: 79.321

7.  CD8(+) T-cell responses to adeno-associated virus capsid in humans.

Authors:  Federico Mingozzi; Marcela V Maus; Daniel J Hui; Denise E Sabatino; Samuel L Murphy; John E J Rasko; Margaret V Ragni; Catherine S Manno; Jurg Sommer; Haiyan Jiang; Glenn F Pierce; Hildegund C J Ertl; Katherine A High
Journal:  Nat Med       Date:  2007-03-18       Impact factor: 53.440

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.  Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates.

Authors:  Amit C Nathwani; John T Gray; Jenny McIntosh; Catherine Y C Ng; Junfang Zhou; Yunyu Spence; Melanie Cochrane; Elaine Gray; Edward G D Tuddenham; Andrew M Davidoff
Journal:  Blood       Date:  2006-11-07       Impact factor: 22.113

10.  Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration.

Authors:  Francesca Simonelli; Albert M Maguire; Francesco Testa; Eric A Pierce; Federico Mingozzi; Jeannette L Bennicelli; Settimio Rossi; Kathleen Marshall; Sandro Banfi; Enrico M Surace; Junwei Sun; T Michael Redmond; Xiaosong Zhu; Kenneth S Shindler; Gui-Shuang Ying; Carmela Ziviello; Carmela Acerra; J Fraser Wright; Jennifer Wellman McDonnell; Katherine A High; Jean Bennett; Alberto Auricchio
Journal:  Mol Ther       Date:  2009-12-01       Impact factor: 11.454

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  4 in total

Review 1.  Gene Therapy for Hemophilia.

Authors:  Arthur W Nienhuis; Amit C Nathwani; Andrew M Davidoff
Journal:  Mol Ther       Date:  2017-04-11       Impact factor: 11.454

2.  Initial Steps for the Development of a Phage-Mediated Gene Replacement Therapy Using CRISPR-Cas9 Technology.

Authors:  Jordi Yang Zhou; Keittisak Suwan; Amin Hajitou
Journal:  J Clin Med       Date:  2020-05-16       Impact factor: 4.241

Review 3.  Gene therapy in PIDs, hemoglobin, ocular, neurodegenerative, and hemophilia B disorders.

Authors:  Arome Solomon Odiba; Nkwachukwu Oziamara Okoro; Olanrewaju Ayodeji Durojaye; Yanjun Wu
Journal:  Open Life Sci       Date:  2021-05-03       Impact factor: 0.938

Review 4.  Clinical development of gene therapy: results and lessons from recent successes.

Authors:  Sandeep Rp Kumar; David M Markusic; Moanaro Biswas; Katherine A High; Roland W Herzog
Journal:  Mol Ther Methods Clin Dev       Date:  2016-05-25       Impact factor: 6.698

  4 in total

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