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Literature DB >> 23770691

A largely random AAV integration profile after LPLD gene therapy.

Christine Kaeppel¹, Stuart G Beattie, Raffaele Fronza, Richard van Logtenstein, Florence Salmon, Sabine Schmidt, Stephan Wolf, Ali Nowrouzi, Hanno Glimm, Christof von Kalle, Harald Petry, Daniel Gaudet, Manfred Schmidt.

Abstract

The clinical application of adeno-associated virus vectors (AAVs) is limited because of concerns about AAV integration-mediated tumorigenicity. We performed integration-site analysis after AAV1-LPL(S447X) intramuscular injection in five lipoprotein lipase-deficient subjects, revealing random nuclear integration and hotspots in mitochondria. We conclude that AAV integration is potentially safe and that vector breakage and integration may occur from each position of the vector genome. Future viral integration-site analyses should include the mitochondrial genome.

Entities: Mutation Species

Mesh：

Substances：
Lipoprotein Lipase

Year: 2013 PMID： 23770691 DOI： 10.1038/nm.3230

Source DB: PubMed Journal: Nat Med ISSN： 1078-8956 Impact factor: 53.440

25 in total

1. A greedy algorithm for aligning DNA sequences.

Authors: Z Zhang; S Schwartz; L Wagner; W Miller
Journal: J Comput Biol Date: 2000 Feb-Apr Impact factor: 1.479

2. Integration frequency and intermolecular recombination of rAAV vectors in non-human primate skeletal muscle and liver.

Authors: Ali Nowrouzi; Magalie Penaud-Budloo; Christine Kaeppel; Uwe Appelt; Caroline Le Guiner; Philippe Moullier; Christof von Kalle; Richard O Snyder; Manfred Schmidt
Journal: Mol Ther Date: 2012-03-27 Impact factor: 11.454

3. Redefining the common insertion site.

Authors: Xiaolin Wu; Brian T Luke; Shawn M Burgess
Journal: Virology Date: 2005-11-04 Impact factor: 3.616

4. 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

5. Gene delivery to mitochondria by targeting modified adenoassociated virus suppresses Leber's hereditary optic neuropathy in a mouse model.

Authors: Hong Yu; Rajeshwari D Koilkonda; Tsung-Han Chou; Vittorio Porciatti; Sacide S Ozdemir; Vince Chiodo; Sanford L Boye; Shannon E Boye; William W Hauswirth; Alfred S Lewin; John Guy
Journal: Proc Natl Acad Sci U S A Date: 2012-04-20 Impact factor: 11.205

6. The Sequence Alignment/Map format and SAMtools.

Authors: Heng Li; Bob Handsaker; Alec Wysoker; Tim Fennell; Jue Ruan; Nils Homer; Gabor Marth; Goncalo Abecasis; Richard Durbin
Journal: Bioinformatics Date: 2009-06-08 Impact factor: 6.937

7. AAV serotype 2 vectors preferentially integrate into active genes in mice.

Authors: Hiroyuki Nakai; Eugenio Montini; Sally Fuess; Theresa A Storm; Markus Grompe; Mark A Kay
Journal: Nat Genet Date: 2003-07 Impact factor: 38.330

8. DNA palindromes with a modest arm length of greater, similar 20 base pairs are a significant target for recombinant adeno-associated virus vector integration in the liver, muscles, and heart in mice.

Authors: Katsuya Inagaki; Susanna M Lewis; Xiaolin Wu; Congrong Ma; David J Munroe; Sally Fuess; Theresa A Storm; Mark A Kay; Hiroyuki Nakai
Journal: J Virol Date: 2007-08-08 Impact factor: 5.103

9. New genes involved in cancer identified by retroviral tagging.

Authors: Takeshi Suzuki; Haifa Shen; Keiko Akagi; Herbert C Morse; James D Malley; Daniel Q Naiman; Nancy A Jenkins; Neal G Copeland
Journal: Nat Genet Date: 2002-08-19 Impact factor: 38.330

10. Real-time definition of non-randomness in the distribution of genomic events.

Authors: Ulrich Abel; Annette Deichmann; Cynthia Bartholomae; Kerstin Schwarzwaelder; Hanno Glimm; Steven Howe; Adrian Thrasher; Alexandrine Garrigue; Salima Hacein-Bey-Abina; Marina Cavazzana-Calvo; Alain Fischer; Dirk Jaeger; Christof von Kalle; Manfred Schmidt
Journal: PLoS One Date: 2007-06-27 Impact factor: 3.240

70 in total

1. Adeno-associated Vector Toxicity-To Be or Not to Be?

Authors: Hildegard Büning; Manfred Schmidt
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Review 2. The potential of adeno-associated viral vectors for gene delivery to muscle tissue.

Authors: Dan Wang; Li Zhong; M Abu Nahid; Guangping Gao
Journal: Expert Opin Drug Deliv Date: 2014-01-03 Impact factor: 6.648

3. Improving lipoprotein profiles by liver-directed gene transfer of low density lipoprotein receptor gene in hypercholesterolaemia mice.

Authors: Hailong Ou; Qinghai Zhang; Jia Zeng
Journal: J Genet Date: 2016-06 Impact factor: 1.166

A largely random AAV integration profile after LPLD gene therapy.

1. A greedy algorithm for aligning DNA sequences.

2. Integration frequency and intermolecular recombination of rAAV vectors in non-human primate skeletal muscle and liver.

3. Redefining the common insertion site.

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

5. Gene delivery to mitochondria by targeting modified adenoassociated virus suppresses Leber's hereditary optic neuropathy in a mouse model.

6. The Sequence Alignment/Map format and SAMtools.

7. AAV serotype 2 vectors preferentially integrate into active genes in mice.

8. DNA palindromes with a modest arm length of greater, similar 20 base pairs are a significant target for recombinant adeno-associated virus vector integration in the liver, muscles, and heart in mice.

9. New genes involved in cancer identified by retroviral tagging.

10. Real-time definition of non-randomness in the distribution of genomic events.

1. Adeno-associated Vector Toxicity-To Be or Not to Be?

Review 2. The potential of adeno-associated viral vectors for gene delivery to muscle tissue.

3. Improving lipoprotein profiles by liver-directed gene transfer of low density lipoprotein receptor gene in hypercholesterolaemia mice.

Review 4. Gene therapy for hemophilia: what does the future hold?

Review 5. Intratympanic Gene Delivery of Antimicrobial Molecules in Otitis Media.

6. Short DNA Hairpins Compromise Recombinant Adeno-Associated Virus Genome Homogeneity.

Review 7. Adeno-associated virus-mediated microRNA delivery and therapeutics.

Review 8. State-of-the-art human gene therapy: part I. Gene delivery technologies.

9. Homologous Recombination-Based Genome Editing by Clade F AAVs Is Inefficient in the Absence of a Targeted DNA Break.

Review 10. Adeno-associated viral vectors for the treatment of hemophilia.