Literature DB >> 23808604

Lessons learned from the clinical development and market authorization of Glybera.

Laura M Bryant1, Devin M Christopher, April R Giles, Christian Hinderer, Jesse L Rodriguez, Jenessa B Smith, Elizabeth A Traxler, Josh Tycko, Adam P Wojno, James M Wilson.   

Abstract

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Year:  2013        PMID： 23808604      PMCID： PMC3992977          DOI： 10.1089/humc.2013.087

Source DB:  PubMed          Journal:  Hum Gene Ther Clin Dev        ISSN： 2324-8637            Impact factor:   5.032


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  18 in total

1.  ADENOVIRUS-ASSOCIATED DEFECTIVE VIRUS PARTICLES.

Authors:  R W ATCHISON; B C CASTO; W M HAMMON
Journal:  Science       Date:  1965-08-13       Impact factor: 47.728

Review 2.  Lipoprotein lipase: structure, function, regulation, and role in disease.

Authors:  James R Mead; Scott A Irvine; Dipak P Ramji
Journal:  J Mol Med (Berl)       Date:  2002-10-24       Impact factor: 4.599

3.  From mutation identification to therapy: discovery and origins of the first approved gene therapy in the Western world.

Authors:  John J P Kastelein; Colin J D Ross; Michael R Hayden
Journal:  Hum Gene Ther       Date:  2013-05       Impact factor: 5.695

4.  Complete rescue of lipoprotein lipase-deficient mice by somatic gene transfer of the naturally occurring LPLS447X beneficial mutation.

Authors:  Colin J D Ross; Guoqing Liu; Jan Albert Kuivenhoven; Jaap Twisk; Jaap Rip; Willemijn van Dop; Katherine J D Ashbourne Excoffon; Suzanne M E Lewis; John J Kastelein; Michael R Hayden
Journal:  Arterioscler Thromb Vasc Biol       Date:  2005-07-07       Impact factor: 8.311

5.  Gene therapy for lipoprotein lipase deficiency: working toward clinical application.

Authors:  Jaap Rip; Melchior C Nierman; Jeroen A Sierts; Wilma Petersen; Karin Van den Oever; Daniel Van Raalte; Colin J D Ross; Michael R Hayden; Andrew C Bakker; Paul Dijkhuizen; Wim T Hermens; Jaap Twisk; Erik Stroes; John J P Kastelein; Jan Albert Kuivenhoven; Janneke M Meulenberg
Journal:  Hum Gene Ther       Date:  2005-11       Impact factor: 5.695

6.  Gene therapy vectors based on adeno-associated virus type 1.

Authors:  W Xiao; N Chirmule; S C Berta; B McCullough; G Gao; J M Wilson
Journal:  J Virol       Date:  1999-05       Impact factor: 5.103

7.  Nucleotide sequence of human cDNA coding for a lipoprotein lipase (LPL) cloned from placental cDNA library.

Authors:  T Gotoda; M Senda; T Gamou; Y Furuichi; K Oka
Journal:  Nucleic Acids Res       Date:  1989-03-25       Impact factor: 16.971

8.  Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer.

Authors:  Federico Mingozzi; Yi-Lin Liu; Eric Dobrzynski; Antje Kaufhold; Jian Hua Liu; YuQin Wang; Valder R Arruda; Katherine A High; Roland W Herzog
Journal:  J Clin Invest       Date:  2003-05       Impact factor: 14.808

9.  Long-term correction of murine lipoprotein lipase deficiency with AAV1-mediated gene transfer of the naturally occurring LPL(S447X) beneficial mutation.

Authors:  Colin J D Ross; Jaap Twisk; Janneke M Meulenberg; Guoqing Liu; Karin van den Oever; Ewoud Moraal; Wim T Hermens; Jaap Rip; John J P Kastelein; Jan Albert Kuivenhoven; Michael R Hayden
Journal:  Hum Gene Ther       Date:  2004-09       Impact factor: 5.695

10.  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
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  55 in total

Review 1.  The potential of adeno-associated viral vectors for gene delivery to muscle tissue.

Authors:  Dan Wang; Li Zhong; M Abu Nahid; Guangping Gao
Journal:  Expert Opin Drug Deliv       Date:  2014-01-03       Impact factor: 6.648

2.  Birth of a new therapeutic platform: 47 years of adeno-associated virus biology from virus discovery to licensed gene therapy.

Authors:  Terence R Flotte
Journal:  Mol Ther       Date:  2013-11       Impact factor: 11.454

3.  AAV vectors expressing LDLR gain-of-function variants demonstrate increased efficacy in mouse models of familial hypercholesterolemia.

Authors:  Suryanarayan Somanathan; Frank Jacobs; Qiang Wang; Alexandra L Hanlon; James M Wilson; Daniel J Rader
Journal:  Circ Res       Date:  2014-07-14       Impact factor: 17.367

Review 4.  AAV: An Overview of Unanswered Questions.

Authors:  Kenneth I Berns; Nicholas Muzyczka
Journal:  Hum Gene Ther       Date:  2017-04       Impact factor: 5.695

Review 5.  Emerging strategies of targeting lipoprotein lipase for metabolic and cardiovascular diseases.

Authors:  Werner J Geldenhuys; Li Lin; Altaf S Darvesh; Prabodh Sadana
Journal:  Drug Discov Today       Date:  2016-10-19       Impact factor: 7.851

Review 6.  Adeno-associated virus-mediated microRNA delivery and therapeutics.

Authors:  Jun Xie; Daniel Robert Burt; Guangping Gao
Journal:  Semin Liver Dis       Date:  2015-01-29       Impact factor: 6.115

7.  Establishment of two quantitative nested qPCR assays targeting the human EPO transgene.

Authors:  E W I Neuberger; I Perez; C Le Guiner; D Moser; T Ehlert; M Allais; P Moullier; P Simon; R O Snyder
Journal:  Gene Ther       Date:  2016-01-11       Impact factor: 5.250

8.  Establishment of a novel cell line for the enhanced production of recombinant adeno-associated virus vectors for gene therapy.

Authors:  Stifani Satkunanathan; Jun Wheeler; Robin Thorpe; Yuan Zhao
Journal:  Hum Gene Ther       Date:  2014-09-11       Impact factor: 5.695

9.  The special case of gene therapy pricing.

Authors:  Troyen A Brennan; James M Wilson
Journal:  Nat Biotechnol       Date:  2014-09       Impact factor: 54.908

Review 10.  Virus-Derived Peptides for Clinical Applications.

Authors:  Mingying Yang; Kegan Sunderland; Chuanbin Mao
Journal:  Chem Rev       Date:  2017-07-19       Impact factor: 60.622
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