Literature DB >> 18418418

Manufacturing and characterizing AAV-based vectors for use in clinical studies.

J F Wright1.   

Abstract

Recombinant adeno-associated virus (AAV)-based vectors expressing therapeutic gene products have shown great promise for human gene therapy. A major challenge for translation of promising research to clinical development is the manufacture and certification of AAV vectors for clinical use. This review summarizes relevant aspects of current Good Manufacturing Practice, focusing on considerations and challenges specific for recombinant AAV.

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Year:  2008        PMID： 18418418     DOI： 10.1038/gt.2008.65

Source DB:  PubMed          Journal:  Gene Ther        ISSN： 0969-7128            Impact factor:   5.250


  58 in total

1.  Targeting Age-Related Neurodegenerative Diseases by AAV-Mediated Gene Therapy.

Authors:  Umut Cagin
Journal:  Adv Exp Med Biol       Date:  2021       Impact factor: 2.622

Review 2.  Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges.

Authors:  Federico Mingozzi; Katherine A High
Journal:  Nat Rev Genet       Date:  2011-05       Impact factor: 53.242

3.  Undetectable transcription of cap in a clinical AAV vector: implications for preformed capsid in immune responses.

Authors:  Bernd Hauck; Samuel L Murphy; Peter H Smith; Guang Qu; Xingge Liu; Olga Zelenaia; Federico Mingozzi; Jürg M Sommer; Katherine A High; J Fraser Wright
Journal:  Mol Ther       Date:  2008-10-21       Impact factor: 11.454

4.  AAV-PHP.B Administration Results in a Differential Pattern of CNS Biodistribution in Non-human Primates Compared with Mice.

Authors:  William A Liguore; Jacqueline S Domire; Dana Button; Yun Wang; Brett D Dufour; Sathya Srinivasan; Jodi L McBride
Journal:  Mol Ther       Date:  2019-08-05       Impact factor: 11.454

Review 5.  Large-scale recombinant adeno-associated virus production.

Authors:  Robert M Kotin
Journal:  Hum Mol Genet       Date:  2011-04-29       Impact factor: 6.150

6.  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

7.  Quantification of AAV particle titers by infrared fluorescence scanning of coomassie-stained sodium dodecyl sulfate-polyacrylamide gels.

Authors:  Erik Kohlbrenner; Els Henckaerts; Kleopatra Rapti; Ronald E Gordon; R Michael Linden; Roger J Hajjar; Thomas Weber
Journal:  Hum Gene Ther Methods       Date:  2012-07-20       Impact factor: 2.396

8.  Site-Specific Post-translational Surface Modification of Adeno-Associated Virus Vectors Using Leucine Zippers.

Authors:  Nicole N Thadani; Joanna Yang; Buhle Moyo; Ciaran M Lee; Maria Y Chen; Gang Bao; Junghae Suh
Journal:  ACS Synth Biol       Date:  2020-02-18       Impact factor: 5.110

9.  Gene therapy in a humanized mouse model of familial hypercholesterolemia leads to marked regression of atherosclerosis.

Authors:  Sadik H Kassim; Hui Li; Luk H Vandenberghe; Christian Hinderer; Peter Bell; Dawn Marchadier; Aisha Wilson; Debra Cromley; Valeska Redon; Hongwei Yu; James M Wilson; Daniel J Rader
Journal:  PLoS One       Date:  2010-10-19       Impact factor: 3.240

10.  The Jeremiah Metzger Lecture: gene therapy for inherited disorders: from Christmas disease to Leber's amaurosis.

Authors:  Katherine A High
Journal:  Trans Am Clin Climatol Assoc       Date:  2009
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