A largely random AAV integration profile after LPLD gene therapy | Zendy

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

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A largely random AAV integration profile after LPLD gene therapy

Author(s) -

Christine Kaeppel,

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

Publication year - 2013

Publication title -

nature medicine

Language(s) - English

Resource type - Journals

SCImago Journal Rank - 19.536

H-Index - 547

eISSN - 1546-170X

pISSN - 1078-8956

DOI - 10.1038/nm.3230

Subject(s) - vector (molecular biology) , genetic enhancement , genome , viral vector , biology , gene , virology , computational biology , genetics , recombinant dna

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.

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