Literature DB >> 23578007

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

John J P Kastelein1, Colin J D Ross, Michael R Hayden.   

Abstract

Mesh：

Year:  2013        PMID： 23578007     DOI： 10.1089/hum.2013.063

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


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  16 in total

1.  [Computer experience and further developments in the respiratory function laboratory (author's transl)].

Authors:  R Schindl; K Mayer; K Aigner
Journal:  Med Klin       Date:  1975-11-07

2.  OneBac: platform for scalable and high-titer production of adeno-associated virus serotype 1-12 vectors for gene therapy.

Authors:  Mario Mietzsch; Sabrina Grasse; Catherine Zurawski; Stefan Weger; Antonette Bennett; Mavis Agbandje-McKenna; Nicholas Muzyczka; Sergei Zolotukhin; Regine Heilbronn
Journal:  Hum Gene Ther       Date:  2014-01-23       Impact factor: 5.695

Review 3.  Lessons learned from the clinical development and market authorization of Glybera.

Authors:  Laura M Bryant; 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
Journal:  Hum Gene Ther Clin Dev       Date:  2013-06-29       Impact factor: 5.032

Review 4.  Virotherapy: cancer gene therapy at last?

Authors:  Alan E Bilsland; Pavlina Spiliopoulou; T R Jeffry Evans
Journal:  F1000Res       Date:  2016-08-30

5.  OneBac 2.0: Sf9 Cell Lines for Production of AAV5 Vectors with Enhanced Infectivity and Minimal Encapsidation of Foreign DNA.

Authors:  Mario Mietzsch; Vincent Casteleyn; Stefan Weger; Sergei Zolotukhin; Regine Heilbronn
Journal:  Hum Gene Ther       Date:  2015-08-06       Impact factor: 5.695

Review 6.  Parvovirus glycan interactions.

Authors:  Lin-Ya Huang; Sujata Halder; Mavis Agbandje-McKenna
Journal:  Curr Opin Virol       Date:  2014-07-19       Impact factor: 7.090

Review 7.  Gene therapy for inherited muscle diseases: where genetics meets rehabilitation medicine.

Authors:  Robynne Braun; Zejing Wang; David L Mack; Martin K Childers
Journal:  Am J Phys Med Rehabil       Date:  2014-11       Impact factor: 2.159

Review 8.  Emptying the stores: lysosomal diseases and therapeutic strategies.

Authors:  Frances M Platt
Journal:  Nat Rev Drug Discov       Date:  2017-11-17       Impact factor: 84.694

9.  Identification and Validation of Small Molecules That Enhance Recombinant Adeno-associated Virus Transduction following High-Throughput Screens.

Authors:  Sarah C Nicolson; Chengwen Li; Matthew L Hirsch; Vincent Setola; R Jude Samulski
Journal:  J Virol       Date:  2016-07-27       Impact factor: 5.103

10.  Re-examination of regulatory opinions in Europe: possible contribution for the approval of the first gene therapy product Glybera.

Authors:  Natsumi Watanabe; Kazuo Yano; Kenichiro Tsuyuki; Teruo Okano; Masayuki Yamato
Journal:  Mol Ther Methods Clin Dev       Date:  2015-02-11       Impact factor: 6.698
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