Concepedia

IgG-cleaving endopeptidase enables in vivo gene therapy in the presence of anti-AAV neutralizing antibodies

Christian Leborgne, Elena Barbon, J M Alexander, Hayley A. Hanby, Sandrine Delignat, Daniel M. Cohen, Fanny Collaud, Saghana Muraleetharan, Dan Lupo, Joseph Silverberg,

Nature Medicine · 2020 · 319 citations · 26 references

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26

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

Jerry R. Mendell, Samiah Al-Zaidy, Richard Shell et al. · New England Journal of Medicine · 2017 · 2.3K 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

, , +9

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

, , +15

Prevalence of Serum IgG and Neutralizing Factors Against Adeno-Associated Virus (AAV) Types 1, 2, 5, 6, 8, and 9 in the Healthy Population: Implications for Gene Therapy Using AAV Vectors

Sylvie Boutin, Virginie Monteilhet, P. Véron et al. · Human Gene Therapy · 2010 · 947 citations

, , +15

Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant

Lindsey A. George, Spencer K. Sullivan, Adam Giermasz et al. · New England Journal of Medicine · 2017 · 759 citations · Full text

, , +15