Concepedia

Non-clinical Safety and Efficacy of an AAV2/8 Vector Administered Intravenously for Treatment of Mucopolysaccharidosis Type VI

Rita Ferla, Marialuisa Alliegro, Jean‐Brice Marteau, Margherita Dell’Anno, Edoardo Nusco, Séverine Pouillot, Stefania Galimberti, Maria Grazia Valsecchi, Vincent Zuliani, Alberto Auricchio

Molecular Therapy — Methods & Clinical Development · 2017 · 50 citations · 47 references

DOIFull text

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Concepts

Abstract

and pave the way for a phase I/II clinical trial based on intravascular infusions of AAV8 in patients with MPS VI.

References

47

Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis

Albert M. Maguire, Francesca Simonelli, Eric A. Pierce et al. · New England Journal of Medicine · 2008 · 2.1K citations · Full text

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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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Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B

Amit C. Nathwani, Edward G. D. Tuddenham, Savita Rangarajan et al. · New England Journal of Medicine · 2011 · 1.8K citations · Full text

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Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

Amit Nathwani, Ulreke M. Reiss, Edward G. D. Tuddenham et al. · New England Journal of Medicine · 2014 · 1.3K citations · Full text

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Disruption of overlapping transcripts in the ROSA βgeo 26 gene trap strain leads to widespread expression of β-galactosidase in mouse embryos and hematopoietic cells

Brian Zambrowicz, Akira Imamoto, Steve Fiering et al. · Proceedings of the National Academy of Sciences · 1997 · 871 citations

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