Literature DB >> 18480442

Biodistribution and safety profile of recombinant adeno-associated virus serotype 6 vectors following intravenous delivery.

Daniel Stone1, Ying Liu, Zong-Yi Li, Robert Strauss, Eric E Finn, James M Allen, Jeffrey S Chamberlain, André Lieber.   

Abstract

Recombinant adeno-associated virus vectors based on serotype 6 (rAAV6) efficiently transduce skeletal muscle after intravenous administration and have shown efficacy in the mdx model of muscular dystrophy. As a prelude to future clinical studies, we investigated the biodistribution and safety profile of rAAV6 in mice. Although it was present in all organs tested, rAAV6 was sequestered mainly in the liver and spleen. rAAV6 had a minimal effect on circulating blood cells and caused no apparent hepatotoxicity or coagulation activation. rAAV6 caused some neutrophil infiltration into the liver, with a transient elevation in cytokine and chemokine transcription/secretion. In summary, rAAV6 induces transient toxicity that subsides almost completely within 72 h and causes no significant side effects.

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Year:  2008        PMID： 18480442      PMCID： PMC2493321          DOI： 10.1128/JVI.00542-08

Source DB:  PubMed          Journal:  J Virol        ISSN： 0022-538X            Impact factor:   5.103


  38 in total

Review 1.  New recombinant serotypes of AAV vectors.

Authors:  Guangping Gao; Luk H Vandenberghe; James M Wilson
Journal:  Curr Gene Ther       Date:  2005-06       Impact factor: 4.391

Review 2.  Adeno-associated virus serotypes: vector toolkit for human gene therapy.

Authors:  Zhijian Wu; Aravind Asokan; R Jude Samulski
Journal:  Mol Ther       Date:  2006-07-07       Impact factor: 11.454

3.  Widespread dispersion of adeno-associated virus serotype 1 and adeno-associated virus serotype 6 vectors in the rat central nervous system and in human glioblastoma multiforme xenografts.

Authors:  Peter C Huszthy; Agnete Svendsen; James M Wilson; Robert M Kotin; Per Eystein Lønning; Rolf Bjerkvig; Frank Hoover
Journal:  Hum Gene Ther       Date:  2005-03       Impact factor: 5.695

4.  rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice.

Authors:  Paul Gregorevic; James M Allen; Elina Minami; Michael J Blankinship; Miki Haraguchi; Leonard Meuse; Eric Finn; Marvin E Adams; Stanley C Froehner; Charles E Murry; Jeffrey S Chamberlain
Journal:  Nat Med       Date:  2006-07-02       Impact factor: 53.440

5.  High-level transgene expression in nonhuman primate liver with novel adeno-associated virus serotypes containing self-complementary genomes.

Authors:  Guang-Ping Gao; You Lu; Xun Sun; Julie Johnston; Roberto Calcedo; Rebecca Grant; James M Wilson
Journal:  J Virol       Date:  2006-06       Impact factor: 5.103

6.  Adeno-associated virus types 5 and 6 use distinct receptors for cell entry.

Authors:  Michael P Seiler; A Dusty Miller; Joseph Zabner; Christine L Halbert
Journal:  Hum Gene Ther       Date:  2006-01       Impact factor: 5.695

7.  Recombinant adenoviruses with large deletions generated by Cre-mediated excision exhibit different biological properties compared with first-generation vectors in vitro and in vivo.

Authors:  A Lieber; C Y He; I Kirillova; M A Kay
Journal:  J Virol       Date:  1996-12       Impact factor: 5.103

8.  Adenovirus binding to blood factors results in liver cell infection and hepatotoxicity.

Authors:  Dmitry M Shayakhmetov; Anuj Gaggar; Shaoheng Ni; Zong-Yi Li; André Lieber
Journal:  J Virol       Date:  2005-06       Impact factor: 5.103

9.  Prevalence of neutralizing antibodies against adeno-associated virus (AAV) types 2, 5, and 6 in cystic fibrosis and normal populations: Implications for gene therapy using AAV vectors.

Authors:  Christine L Halbert; A Dusty Miller; Sharon McNamara; Julia Emerson; Ronald L Gibson; Bonnie Ramsey; Moira L Aitken
Journal:  Hum Gene Ther       Date:  2006-04       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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  10 in total

1.  Expression of dog microdystrophin in mouse and dog muscles by gene therapy.

Authors:  Christophe Pichavant; Pierre Chapdelaine; Daniel G Cerri; Jean-Christophe Dominique; Simon P Quenneville; Daniel Skuk; Joe N Kornegay; João Cs Bizario; Xiao Xiao; Jacques P Tremblay
Journal:  Mol Ther       Date:  2010-02-23       Impact factor: 11.454

2.  Biodistribution of AAV8 vectors expressing human low-density lipoprotein receptor in a mouse model of homozygous familial hypercholesterolemia.

Authors:  Shu-Jen Chen; Julio Sanmiguel; Martin Lock; Deirdre McMenamin; Christine Draper; Maria P Limberis; Sadik H Kassim; Suryanarayan Somanathan; Peter Bell; Julie C Johnston; Daniel J Rader; James M Wilson
Journal:  Hum Gene Ther Clin Dev       Date:  2013-11-09       Impact factor: 5.032

Review 3.  Promise of adeno-associated virus as a gene therapy vector for cardiovascular diseases.

Authors:  Abesh Bera; Dwaipayan Sen
Journal:  Heart Fail Rev       Date:  2017-11       Impact factor: 4.214

4.  Human galectin 3 binding protein interacts with recombinant adeno-associated virus type 6.

Authors:  Jerome Denard; Cyriaque Beley; Robert Kotin; René Lai-Kuen; Stéphane Blot; Hervé Leh; Aravind Asokan; R Jude Samulski; Philippe Moullier; Thomas Voit; Luis Garcia; Fedor Svinartchouk
Journal:  J Virol       Date:  2012-04-11       Impact factor: 5.103

5.  Therapeutic impact of systemic AAV-mediated RNA interference in a mouse model of myotonic dystrophy.

Authors:  Darren R Bisset; Ewa A Stepniak-Konieczna; Maja Zavaljevski; Jessica Wei; Gregory T Carter; Michael D Weiss; Joel R Chamberlain
Journal:  Hum Mol Genet       Date:  2015-06-16       Impact factor: 6.150

6.  Immuno-therapy with anti-CTLA4 antibodies in tolerized and non-tolerized mouse tumor models.

Authors:  Jonas Persson; Ines Beyer; Roma Yumul; ZongYi Li; Hans-Peter Kiem; Steve Roffler; André Lieber
Journal:  PLoS One       Date:  2011-07-14       Impact factor: 3.240

7.  Biodistribution of adeno-associated virus type 2 carrying multi-characteristic opsin in dogs following intravitreal injection.

Authors:  Kissaou T Tchedre; Subrata Batabyal; Melissa Galicia; Darryl Narcisse; Sourajit Mitra Mustafi; Ananta Ayyagari; Sai Chavala; Samarendra K Mohanty
Journal:  J Cell Mol Med       Date:  2021-08-21       Impact factor: 5.310

8.  A pilot study of neonatal GALT gene replacement using AAV9 dramatically lowers galactose metabolites in blood, liver, and brain and minimizes cataracts in GALT-null rat pups.

Authors:  Shauna A Rasmussen; Jennifer M I Daenzer; Judith L Fridovich-Keil
Journal:  J Inherit Metab Dis       Date:  2020-09-17       Impact factor: 4.982

9.  Screening for gene doping transgenes in horses via the use of massively parallel sequencing.

Authors:  Jillian Maniego; Bogusia Pesko; Jocelyn Habershon-Butcher; Jim Huggett; Polly Taylor; James Scarth; Edward Ryder
Journal:  Gene Ther       Date:  2021-07-19       Impact factor: 5.250

10.  Characterization of hepatic macrophages and evaluation of inflammatory response in heme oxygenase-1 deficient mice exposed to scAAV9 vectors.

Authors:  Mateusz Tomczyk; Izabela Kraszewska; Robert Mąka; Agnieszka Waligórska; Józef Dulak; Agnieszka Jaźwa-Kusior
Journal:  PLoS One       Date:  2020-10-15       Impact factor: 3.240
  10 in total

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