Literature DB >> 19861950

Systematic evaluation of AAV vectors for liver directed gene transfer in murine models.

Lili Wang1, Huan Wang, Peter Bell, Robert J McCarter, Jianping He, Roberto Calcedo, Luk H Vandenberghe, Hiroki Morizono, Mark L Batshaw, James M Wilson.   

Abstract

Vectors based on adeno-associated viruses (AAVs) are being evaluated for use in liver-directed gene therapy. Candidates have been preselected on the basis of capsid structure that plays an important role in determining performance profiles. We describe a comprehensive and statistically powered set of mouse studies designed to compare the performance of vectors based on seven novel AAV capsids. The key criteria used to select candidates for successful gene therapy are high level and stable transgene expression in the absence of toxicity. Based on these criteria, the best performing vectors, AAV8, AAVhu.37, and AAVrh.8, will be further evaluated in nonhuman primates (NHPs).

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Year:  2009        PMID： 19861950      PMCID： PMC2839210          DOI： 10.1038/mt.2009.246

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


  24 in total

1.  A pilot study of in vivo liver-directed gene transfer with an adenoviral vector in partial ornithine transcarbamylase deficiency.

Authors:  Steven E Raper; Marc Yudkoff; Narendra Chirmule; Guang-Ping Gao; Fred Nunes; Ziv J Haskal; Emma E Furth; Kathleen J Propert; Michael B Robinson; Susan Magosin; Heather Simoes; Lisa Speicher; Joseph Hughes; John Tazelaar; Nelson A Wivel; James M Wilson; Mark L Batshaw
Journal:  Hum Gene Ther       Date:  2002-01-01       Impact factor: 5.695

2.  Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors.

Authors:  R O Snyder; C H Miao; G A Patijn; S K Spratt; O Danos; D Nagy; A M Gown; B Winther; L Meuse; L K Cohen; A R Thompson; M A Kay
Journal:  Nat Genet       Date:  1997-07       Impact factor: 38.330

3.  Biology of AAV serotype vectors in liver-directed gene transfer to nonhuman primates.

Authors:  Guangping Gao; You Lu; Roberto Calcedo; Rebecca L Grant; Peter Bell; Lili Wang; Joanita Figueredo; Martin Lock; James M Wilson
Journal:  Mol Ther       Date:  2005-10-10       Impact factor: 11.454

4.  Comparison of the ability of adeno-associated viral vectors pseudotyped with serotype 2, 5, and 8 capsid proteins to mediate efficient transduction of the liver in murine and nonhuman primate models.

Authors:  Andrew M Davidoff; John T Gray; Catherine Y C Ng; Youbin Zhang; Junfang Zhou; Yunyu Spence; Yusura Bakar; Amit C Nathwani
Journal:  Mol Ther       Date:  2005-06       Impact factor: 11.454

5.  Human immunoglobulin inhibits liver transduction by AAV vectors at low AAV2 neutralizing titers in SCID mice.

Authors:  Ciaran D Scallan; Haiyan Jiang; Tongyao Liu; Susannah Patarroyo-White; Jurg M Sommer; Shangzhen Zhou; Linda B Couto; Glenn F Pierce
Journal:  Blood       Date:  2005-10-25       Impact factor: 22.113

6.  Sustained correction of disease in naive and AAV2-pretreated hemophilia B dogs: AAV2/8-mediated, liver-directed gene therapy.

Authors:  Lili Wang; Roberto Calcedo; Timothy C Nichols; Dwight A Bellinger; Aaron Dillow; Inder M Verma; James M Wilson
Journal:  Blood       Date:  2005-01-06       Impact factor: 22.113

7.  Efficacy of an adeno-associated virus 8-pseudotyped vector in glycogen storage disease type II.

Authors:  Baodong Sun; Haoyue Zhang; Luis M Franco; Sarah P Young; Ayn Schneider; Andrew Bird; Andrea Amalfitano; Y-T Chen; Dwight D Koeberl
Journal:  Mol Ther       Date:  2005-01       Impact factor: 11.454

8.  Total correction of hemophilia A mice with canine FVIII using an AAV 8 serotype.

Authors:  Rita Sarkar; Renee Tetreault; Guangping Gao; Lili Wang; Peter Bell; Randy Chandler; James M Wilson; Haig H Kazazian
Journal:  Blood       Date:  2003-10-09       Impact factor: 22.113

9.  Clades of Adeno-associated viruses are widely disseminated in human tissues.

Authors:  Guangping Gao; Luk H Vandenberghe; Mauricio R Alvira; You Lu; Roberto Calcedo; Xiangyang Zhou; James M Wilson
Journal:  J Virol       Date:  2004-06       Impact factor: 5.103

10.  Naturally occurring singleton residues in AAV capsid impact vector performance and illustrate structural constraints.

Authors:  L H Vandenberghe; E Breous; H-J Nam; G Gao; R Xiao; A Sandhu; J Johnston; Z Debyser; M Agbandje-McKenna; J M Wilson
Journal:  Gene Ther       Date:  2009-12       Impact factor: 5.250
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  74 in total

1.  Hepatic gene transfer in neonatal mice by adeno-associated virus serotype 8 vector.

Authors:  Lili Wang; Huan Wang; Peter Bell; Deirdre McMenamin; James M Wilson
Journal:  Hum Gene Ther       Date:  2012-02-08       Impact factor: 5.695

2.  Sustained enzymatic correction by rAAV-mediated liver gene therapy protects against induced motor neuropathy in acute porphyria mice.

Authors:  Carmen Unzu; Ana Sampedro; Itsaso Mauleón; Manuel Alegre; Stuart G Beattie; Rafael Enríquez de Salamanca; Jolanda Snapper; Jaap Twisk; Harald Petry; Gloria González-Aseguinolaza; Julio Artieda; María Sol Rodríguez-Pena; Jesús Prieto; Antonio Fontanellas
Journal:  Mol Ther       Date:  2010-09-28       Impact factor: 11.454

3.  Mapping a neutralizing epitope onto the capsid of adeno-associated virus serotype 8.

Authors:  Brittney L Gurda; Christina Raupp; Ruth Popa-Wagner; Matthias Naumer; Norman H Olson; Robert Ng; Robert McKenna; Timothy S Baker; Jürgen A Kleinschmidt; Mavis Agbandje-McKenna
Journal:  J Virol       Date:  2012-05-16       Impact factor: 5.103

4.  Hepatic miR-181b-5p Contributes to Glycogen Synthesis Through Targeting EGR1.

Authors:  Shuyue Wang; Chen Liang; Huihan Ai; Meiting Yang; Jingwen Yi; Lei Liu; Zhenbo Song; Yongli Bao; Yuxin Li; Luguo Sun; Huiying Zhao
Journal:  Dig Dis Sci       Date:  2019-01-09       Impact factor: 3.199

5.  The pleiotropic effects of natural AAV infections on liver-directed gene transfer in macaques.

Authors:  Lili Wang; Roberto Calcedo; Huan Wang; Peter Bell; Rebecca Grant; Luk H Vandenberghe; Julio Sanmiguel; Hiroki Morizono; Mark L Batshaw; James M Wilson
Journal:  Mol Ther       Date:  2009-11-03       Impact factor: 11.454

6.  The role of apoptosis in immune hyporesponsiveness following AAV8 liver gene transfer.

Authors:  Susan M Faust; Peter Bell; Yanqing Zhu; Julio Sanmiguel; James M Wilson
Journal:  Mol Ther       Date:  2013-10-15       Impact factor: 11.454

7.  Impact of pre-existing immunity on gene transfer to nonhuman primate liver with adeno-associated virus 8 vectors.

Authors:  Lili Wang; Roberto Calcedo; Peter Bell; Jianping Lin; Rebecca L Grant; Don L Siegel; James M Wilson
Journal:  Hum Gene Ther       Date:  2011-06-08       Impact factor: 5.695

8.  Nonclinical Pharmacology/Toxicology Study of AAV8.TBG.mLDLR and AAV8.TBG.hLDLR in a Mouse Model of Homozygous Familial Hypercholesterolemia.

Authors:  Jenny A Greig; Maria P Limberis; Peter Bell; Shu-Jen Chen; Roberto Calcedo; Daniel J Rader; James M Wilson
Journal:  Hum Gene Ther Clin Dev       Date:  2017-03       Impact factor: 5.032

9.  HIV-1 suppression and durable control by combining single broadly neutralizing antibodies and antiretroviral drugs in humanized mice.

Authors:  Joshua A Horwitz; Ariel Halper-Stromberg; Hugo Mouquet; Alexander D Gitlin; Anna Tretiakova; Thomas R Eisenreich; Marine Malbec; Sophia Gravemann; Eva Billerbeck; Marcus Dorner; Hildegard Büning; Olivier Schwartz; Elena Knops; Rolf Kaiser; Michael S Seaman; James M Wilson; Charles M Rice; Alexander Ploss; Pamela J Bjorkman; Florian Klein; Michel C Nussenzweig
Journal:  Proc Natl Acad Sci U S A       Date:  2013-09-16       Impact factor: 11.205

10.  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
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