Literature DB >> 26906613

Genotoxicity in Mice Following AAV Gene Delivery: A Safety Concern for Human Gene Therapy?

Randy J Chandler1, Matthew C LaFave2, Gaurav K Varshney2, Shawn M Burgess2, Charles P Venditti3.   

Abstract

Entities:  

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Year:  2016        PMID： 26906613      PMCID： PMC4817827          DOI： 10.1038/mt.2016.17

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


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  19 in total

1.  Adeno-Associated Virus Type 2 and Hepatocellular Carcinoma?

Authors:  Kenneth I Berns; Barry J Byrne; Terence R Flotte; Guangping Gao; William W Hauswirth; Roland W Herzog; Nicholas Muzyczka; Thierry VandenDriessche; Xiao Xiao; Sergei Zolotukhin; Arun Srivastava
Journal:  Hum Gene Ther       Date:  2015-12       Impact factor: 5.695

2.  Induction of hepatocellular carcinoma by in vivo gene targeting.

Authors:  Pei-Rong Wang; Mei Xu; Sara Toffanin; Yi Li; Josep M Llovet; David W Russell
Journal:  Proc Natl Acad Sci U S A       Date:  2012-06-25       Impact factor: 11.205

3.  Patterns of scAAV vector insertion associated with oncogenic events in a mouse model for genotoxicity.

Authors:  Lucia E Rosas; Jessica L Grieves; Kimberly Zaraspe; Krista Md La Perle; Haiyan Fu; Douglas M McCarty
Journal:  Mol Ther       Date:  2012-09-18       Impact factor: 11.454

4.  Observed incidence of tumorigenesis in long-term rodent studies of rAAV vectors.

Authors:  A Donsante; C Vogler; N Muzyczka; J M Crawford; J Barker; T Flotte; M Campbell-Thompson; T Daly; M S Sands
Journal:  Gene Ther       Date:  2001-09       Impact factor: 5.250

5.  Assessing the potential for AAV vector genotoxicity in a murine model.

Authors:  Hojun Li; Nirav Malani; Shari R Hamilton; Alexander Schlachterman; Giulio Bussadori; Shyrie E Edmonson; Rachel Shah; Valder R Arruda; Federico Mingozzi; J Fraser Wright; Frederic D Bushman; Katherine A High
Journal:  Blood       Date:  2010-11-24       Impact factor: 22.113

6.  Characterization of the mouse adeno-associated virus AAVS1 ortholog.

Authors:  Nathalie Dutheil; Miran Yoon-Robarts; Peter Ward; Els Henckaerts; Lucy Skrabanek; Kenneth I Berns; Fabien Campagne; R Michael Linden
Journal:  J Virol       Date:  2004-08       Impact factor: 5.103

7.  AAV vector integration sites in mouse hepatocellular carcinoma.

Authors:  Anthony Donsante; Daniel G Miller; Yi Li; Carole Vogler; Elizabeth M Brunt; David W Russell; Mark S Sands
Journal:  Science       Date:  2007-07-27       Impact factor: 47.728

8.  A modified sleeping beauty transposon system that can be used to model a wide variety of human cancers in mice.

Authors:  Adam J Dupuy; Laura M Rogers; Jinsil Kim; Kishore Nannapaneni; Timothy K Starr; Pentao Liu; David A Largaespada; Todd E Scheetz; Nancy A Jenkins; Neal G Copeland
Journal:  Cancer Res       Date:  2009-10-06       Impact factor: 12.701

9.  Identification of rtl1, a retrotransposon-derived imprinted gene, as a novel driver of hepatocarcinogenesis.

Authors:  Jesse D Riordan; Vincent W Keng; Barbara R Tschida; Todd E Scheetz; Jason B Bell; Kelly M Podetz-Pedersen; Catherine D Moser; Neal G Copeland; Nancy A Jenkins; Lewis R Roberts; David A Largaespada; Adam J Dupuy
Journal:  PLoS Genet       Date:  2013-04-04       Impact factor: 5.917

10.  Lentiviral vector-based insertional mutagenesis identifies genes associated with liver cancer.

Authors:  Marco Ranzani; Daniela Cesana; Cynthia C Bartholomae; Francesca Sanvito; Mauro Pala; Fabrizio Benedicenti; Pierangela Gallina; Lucia Sergi Sergi; Stefania Merella; Alessandro Bulfone; Claudio Doglioni; Christof von Kalle; Yoon Jun Kim; Manfred Schmidt; Giovanni Tonon; Luigi Naldini; Eugenio Montini
Journal:  Nat Methods       Date:  2013-01-13       Impact factor: 28.547
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  19 in total

1.  Identification of liver-specific enhancer-promoter activity in the 3' untranslated region of the wild-type AAV2 genome.

Authors:  Grant J Logan; Allison P Dane; Claus V Hallwirth; Christine M Smyth; Emilie E Wilkie; Anais K Amaya; Erhua Zhu; Neeta Khandekar; Samantha L Ginn; Sophia H Y Liao; Sharon C Cunningham; Natsuki Sasaki; Martí Cabanes-Creus; Patrick P L Tam; David W Russell; Leszek Lisowski; Ian E Alexander
Journal:  Nat Genet       Date:  2017-06-19       Impact factor: 38.330

2.  Adeno-Associated Virus Genome Interactions Important for Vector Production and Transduction.

Authors:  Anna C Maurer; Matthew D Weitzman
Journal:  Hum Gene Ther       Date:  2020-05       Impact factor: 5.695

Review 3.  Gene Therapy for Methylmalonic Acidemia: Past, Present, and Future.

Authors:  Randy J Chandler; Charles P Venditti
Journal:  Hum Gene Ther       Date:  2019-08-16       Impact factor: 5.695

4.  AAV integration in human hepatocytes.

Authors:  Dhwanil A Dalwadi; Andrea Calabria; Amita Tiyaboonchai; Jeffrey Posey; Willscott E Naugler; Eugenio Montini; Markus Grompe
Journal:  Mol Ther       Date:  2021-08-28       Impact factor: 12.910

5.  Self-Complementary AAV9 Gene Delivery Partially Corrects Pathology Associated with Juvenile Neuronal Ceroid Lipofuscinosis (CLN3).

Authors:  Megan E Bosch; Amy Aldrich; Rachel Fallet; Jessica Odvody; Maria Burkovetskaya; Kaitlyn Schuberth; Julie A Fitzgerald; Kevin D Foust; Tammy Kielian
Journal:  J Neurosci       Date:  2016-09-14       Impact factor: 6.167

6.  Enhanced liver gene transfer and evasion of preexisting humoral immunity with exosome-enveloped AAV vectors.

Authors:  Amine Meliani; Florence Boisgerault; Zachary Fitzpatrick; Solenne Marmier; Christian Leborgne; Fanny Collaud; Marcelo Simon Sola; Severine Charles; Giuseppe Ronzitti; Alban Vignaud; Laetitia van Wittenberghe; Beatrice Marolleau; Fabienne Jouen; Sisareuth Tan; Olivier Boyer; Olivier Christophe; Alain R Brisson; Casey A Maguire; Federico Mingozzi
Journal:  Blood Adv       Date:  2017-10-16

Review 7.  Modulating gene regulation to treat genetic disorders.

Authors:  Navneet Matharu; Nadav Ahituv
Journal:  Nat Rev Drug Discov       Date:  2020-10-05       Impact factor: 84.694

8.  Long-term functional correction of cystathionine β-synthase deficiency in mice by adeno-associated viral gene therapy.

Authors:  Hyung-Ok Lee; Christiana O Salami; Dolan Sondhi; Stephen M Kaminsky; Ronald G Crystal; Warren D Kruger
Journal:  J Inherit Metab Dis       Date:  2021-10-11       Impact factor: 4.750

9.  Enhanced Efficacy and Increased Long-Term Toxicity of CNS-Directed, AAV-Based Combination Therapy for Krabbe Disease.

Authors:  Yedda Li; Christopher A Miller; Lauren K Shea; Xuntian Jiang; Miguel A Guzman; Randy J Chandler; Sai M Ramakrishnan; Stephanie N Smith; Charles P Venditti; Carole A Vogler; Daniel S Ory; Timothy J Ley; Mark S Sands
Journal:  Mol Ther       Date:  2021-01-01       Impact factor: 11.454

10.  Promoterless, Nuclease-Free Genome Editing Confers a Growth Advantage for Corrected Hepatocytes in Mice With Methylmalonic Acidemia.

Authors:  Randy J Chandler; Leah E Venturoni; Jing Liao; Brandon T Hubbard; Jessica L Schneller; Victoria Hoffmann; Susana Gordo; Shengwen Zang; Chih-Wei Ko; Nelson Chau; Kyle Chiang; Mark A Kay; Adi Barzel; Charles P Venditti
Journal:  Hepatology       Date:  2021-05-21       Impact factor: 17.298
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