Concepedia

Hydrostatic isolated limb perfusion with adeno-associated virus vectors enhances correction of skeletal muscle in Pompe disease

Baodong Sun, Shuowei Li, Andrew Bird, D D Koeberl

Gene Therapy · 2010 · 12 citations · 30 references

DOIFull text

Open access

Concepts

References

30

Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

Catherine S. Manno, Glenn F. Pierce, Valder R. Arruda et al. · Nature Medicine · 2006 · 2.1K citations

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Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy

Guangping Gao, Mauricio R. Alvira, Lili Wang et al. · Proceedings of the National Academy of Sciences · 2002 · 1.5K citations · Full text

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Recombinant human acid α-glucosidase

Priya S. Kishnani, Deya Corzo, Marc Nicolino et al. · Neurology · 2006 · 772 citations · Full text

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Systemic delivery of genes to striated muscles using adeno-associated viral vectors

Paul Gregorevic, Michael J. Blankinship, James M. Allen et al. · Nature Medicine · 2004 · 611 citations · Full text

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Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants

Priya S. Kishnani, Paula Goldenberg, Stephanie DeArmey et al. · Molecular Genetics and Metabolism · 2009 · 379 citations

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