Literature DB >> 27727215

Early clinical data raise the bar for hemophilia gene therapies.

Elie Dolgin.   

Abstract

Entities:  

Year:  2016        PMID： 27727215     DOI： 10.1038/nbt1016-999

Source DB:  PubMed          Journal:  Nat Biotechnol        ISSN： 1087-0156            Impact factor:   54.908


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  4 in total

1.  Biogen and UPenn join forces to commercialize gene therapies.

Authors:  Cormac Sheridan
Journal:  Nat Biotechnol       Date:  2016-08-09       Impact factor: 54.908

2.  Factor VIII-Mimetic Function of Humanized Bispecific Antibody in Hemophilia A.

Authors:  Midori Shima; Hideji Hanabusa; Masashi Taki; Tadashi Matsushita; Tetsuji Sato; Katsuyuki Fukutake; Naoki Fukazawa; Koichiro Yoneyama; Hiroki Yoshida; Keiji Nogami
Journal:  N Engl J Med       Date:  2016-05-26       Impact factor: 91.245

3.  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

4.  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
  4 in total
  10 in total

1.  After Glybera's withdrawal, what's next for gene therapy?

Authors:  Melanie Senior
Journal:  Nat Biotechnol       Date:  2017-06-07       Impact factor: 54.908

2.  Delivering efficient liver-directed AAV-mediated gene therapy.

Authors:  J Baruteau; S N Waddington; I E Alexander; P Gissen
Journal:  Gene Ther       Date:  2017-01-12       Impact factor: 5.250

3.  Blood feud erupts over Roche's bispecific antibody for hemophilia.

Authors:  Elie Dolgin
Journal:  Nat Biotechnol       Date:  2017-09-11       Impact factor: 54.908

Review 4.  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

Review 5.  Complexity of immune responses to AAV transgene products - Example of factor IX.

Authors:  Roland W Herzog
Journal:  Cell Immunol       Date:  2017-05-29       Impact factor: 4.868

6.  Protection is not always a good thing: The immune system's impact on gene therapy.

Authors:  Martiela Vaz de Freitas; Lariane Frâncio; Laura Haleva; Ursula da Silveira Matte
Journal:  Genet Mol Biol       Date:  2022-07-15       Impact factor: 2.087

7.  Direct Head-to-Head Evaluation of Recombinant Adeno-associated Viral Vectors Manufactured in Human versus Insect Cells.

Authors:  Oleksandr Kondratov; Damien Marsic; Sean M Crosson; Hector R Mendez-Gomez; Oleksandr Moskalenko; Mario Mietzsch; Regine Heilbronn; Jonathan R Allison; Kari B Green; Mavis Agbandje-McKenna; Sergei Zolotukhin
Journal:  Mol Ther       Date:  2017-08-10       Impact factor: 11.454

8.  Evaluation of engineered AAV capsids for hepatic factor IX gene transfer in murine and canine models.

Authors:  David M Markusic; Timothy C Nichols; Elizabeth P Merricks; Brett Palaschak; Irene Zolotukhin; Damien Marsic; Sergei Zolotukhin; Arun Srivastava; Roland W Herzog
Journal:  J Transl Med       Date:  2017-05-01       Impact factor: 5.531

Review 9.  Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects.

Authors:  Julien Baruteau; Simon N Waddington; Ian E Alexander; Paul Gissen
Journal:  J Inherit Metab Dis       Date:  2017-05-31       Impact factor: 4.982

10.  An Observational Study from Long-Term AAV Re-administration in Two Hemophilia Dogs.

Authors:  Junjiang Sun; Wenwei Shao; Xiaojing Chen; Elizabeth P Merricks; Lauren Wimsey; Yasmina L Abajas; Glenn P Niemeyer; Clinton D Lothrop; Paul E Monahan; R Jude Samulski; Timothy C Nichols; Chengwen Li
Journal:  Mol Ther Methods Clin Dev       Date:  2018-08-04       Impact factor: 6.698
  10 in total

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