Literature DB >> 21532610

A barrel of monkeys: scAAV8 gene therapy for hemophilia in nonhuman primates.

Randy J Chandler1, Charles P Venditti.   

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Year:  2011        PMID： 21532610      PMCID： PMC3098648          DOI： 10.1038/mt.2011.73

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


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  14 in total

1.  Rapid uncoating of vector genomes is the key to efficient liver transduction with pseudotyped adeno-associated virus vectors.

Authors:  Clare E Thomas; Theresa A Storm; Zan Huang; Mark A Kay
Journal:  J Virol       Date:  2004-03       Impact factor: 5.103

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

3.  Long-term safety and efficacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins.

Authors:  Amit C Nathwani; Cecilia Rosales; Jenny McIntosh; Ghasem Rastegarlari; Devhrut Nathwani; Deepak Raj; Sushmita Nawathe; Simon N Waddington; Roderick Bronson; Scott Jackson; Robert E Donahue; Katherine A High; Federico Mingozzi; Catherine Y C Ng; Junfang Zhou; Yunyu Spence; M Beth McCarville; Marc Valentine; James Allay; John Coleman; Susan Sleep; John T Gray; Arthur W Nienhuis; Andrew M Davidoff
Journal:  Mol Ther       Date:  2011-01-18       Impact factor: 11.454

4.  AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B.

Authors:  Catherine S Manno; Amy J Chew; Sylvia Hutchison; Peter J Larson; Roland W Herzog; Valder R Arruda; Shing Jen Tai; Margaret V Ragni; Arthur Thompson; Margareth Ozelo; Linda B Couto; Debra G B Leonard; Frederick A Johnson; Alan McClelland; Ciaran Scallan; Erik Skarsgard; Alan W Flake; Mark A Kay; Katherine A High; Bertil Glader
Journal:  Blood       Date:  2002-12-19       Impact factor: 22.113

5.  AAV vector integration sites in mouse hepatocellular carcinoma.

Authors:  Anthony Donsante; Daniel G Miller; Yi Li; Carole Vogler; Elizabeth M Brunt; David W Russell; Mark S Sands
Journal:  Science       Date:  2007-07-27       Impact factor: 47.728

6.  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

7.  Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy.

Authors:  Guang-Ping Gao; Mauricio R Alvira; Lili Wang; Roberto Calcedo; Julie Johnston; James M Wilson
Journal:  Proc Natl Acad Sci U S A       Date:  2002-08-21       Impact factor: 11.205

8.  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

9.  Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver.

Authors:  Amit C Nathwani; John T Gray; Catherine Y C Ng; Junfang Zhou; Yunyu Spence; Simon N Waddington; Edward G D Tuddenham; Geoffrey Kemball-Cook; Jenny McIntosh; Mariette Boon-Spijker; Koen Mertens; Andrew M Davidoff
Journal:  Blood       Date:  2005-12-01       Impact factor: 22.113

Review 10.  Gene therapy for haemophilia.

Authors:  Samuel L Murphy; Katherine A High
Journal:  Br J Haematol       Date:  2008-03       Impact factor: 6.998
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  2 in total

1.  Minimizing the inhibitory effect of neutralizing antibody for efficient gene expression in the liver with adeno-associated virus 8 vectors.

Authors:  Jun Mimuro; Hiroaki Mizukami; Shuji Hishikawa; Tomokazu Ikemoto; Akira Ishiwata; Asuka Sakata; Tsukasa Ohmori; Seiji Madoiwa; Fumiko Ono; Keiya Ozawa; Yoichi Sakata
Journal:  Mol Ther       Date:  2012-12-18       Impact factor: 11.454

2.  Mini-review on "A novel one-step purification of mouse factor IX".

Authors:  Sumita Choudhury; William E Plautz; Cosette Zacarias; Rinku Majumder
Journal:  J Rare Dis Res Treat       Date:  2016
  2 in total

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