Literature DB >> 25397929

Our journey to successful gene therapy for hemophilia B.

Amit C Nathwani1, Arthur W Nienhuis, Andrew M Davidoff.   

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Year:  2014        PMID： 25397929      PMCID： PMC4236090          DOI： 10.1089/hum.2014.2540

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


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  33 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.  Design and packaging of adeno-associated virus gene targeting vectors.

Authors:  R K Hirata; D W Russell
Journal:  J Virol       Date:  2000-05       Impact factor: 5.103

4.  Improved muscle-derived expression of human coagulation factor IX from a skeletal actin/CMV hybrid enhancer/promoter.

Authors:  J N Hagstrom; L B Couto; C Scallan; M Burton; M L McCleland; P A Fields; V R Arruda; R W Herzog; K A High
Journal:  Blood       Date:  2000-04-15       Impact factor: 22.113

5.  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

6.  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

7.  Seroepidemiological and ecological studies of the adenovirus-associated satellite viruses.

Authors:  W P Parks; D W Boucher; J L Melnick; L H Taber; M D Yow
Journal:  Infect Immun       Date:  1970-12       Impact factor: 3.441

8.  Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo.

Authors:  D M McCarty; H Fu; P E Monahan; C E Toulson; P Naik; R J Samulski
Journal:  Gene Ther       Date:  2003-12       Impact factor: 5.250

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.  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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  5 in total

Review 1.  Progress and challenges in viral vector manufacturing.

Authors:  Johannes C M van der Loo; J Fraser Wright
Journal:  Hum Mol Genet       Date:  2015-10-30       Impact factor: 6.150

Review 2.  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

3.  Systemic AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in Dogs.

Authors:  David L Mack; Karine Poulard; Melissa A Goddard; Virginie Latournerie; Jessica M Snyder; Robert W Grange; Matthew R Elverman; Jérôme Denard; Philippe Veron; Laurine Buscara; Christine Le Bec; Jean-Yves Hogrel; Annie G Brezovec; Hui Meng; Lin Yang; Fujun Liu; Michael O'Callaghan; Nikhil Gopal; Valerie E Kelly; Barbara K Smith; Jennifer L Strande; Fulvio Mavilio; Alan H Beggs; Federico Mingozzi; Michael W Lawlor; Ana Buj-Bello; Martin K Childers
Journal:  Mol Ther       Date:  2017-02-22       Impact factor: 11.454

Review 4.  Gene Therapy Leaves a Vicious Cycle.

Authors:  Reena Goswami; Gayatri Subramanian; Liliya Silayeva; Isabelle Newkirk; Deborah Doctor; Karan Chawla; Saurabh Chattopadhyay; Dhyan Chandra; Nageswararao Chilukuri; Venkaiah Betapudi
Journal:  Front Oncol       Date:  2019-04-24       Impact factor: 6.244

Review 5.  Current Clinical Applications of In Vivo Gene Therapy with AAVs.

Authors:  Jerry R Mendell; Samiah A Al-Zaidy; Louise R Rodino-Klapac; Kimberly Goodspeed; Steven J Gray; Christine N Kay; Sanford L Boye; Shannon E Boye; Lindsey A George; Stephanie Salabarria; Manuela Corti; Barry J Byrne; Jacques P Tremblay
Journal:  Mol Ther       Date:  2020-12-10       Impact factor: 11.454
  5 in total

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