Concepedia

Development of an In Vitro Biopotency Assay for an AAV8 Hemophilia B Gene Therapy Vector Suitable for Clinical Product Release

Johannes Lengler, Sogué Coulibaly, Bernadette Gruber, Reinhard Ilk, Josef Mayrhofer, Friedrich Scheiflinger, Werner Hoellriegl, Falko G. Falkner, Hanspeter Rottensteiner

Molecular Therapy — Methods & Clinical Development · 2020 · 19 citations · 27 references

DOIFull text

Open access

Concepts

Abstract

assay thus constitutes a viable alternative to using animals for lot release testing.

References

27

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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Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis

DM McCarty, PE Monahan, RJ Samulski · Gene Therapy · 2001 · 725 citations

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Universal Real-Time PCR for the Detection and Quantification of Adeno-Associated Virus Serotype 2-Derived Inverted Terminal Repeat Sequences

Christine Aurnhammer, Maren Haase, Nadine Muether et al. · Human Gene Therapy Methods · 2012 · 490 citations

, , +14

Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver

Amit C. Nathwani, John T. Gray, Catherine Y. Ng et al. · Blood · 2005 · 422 citations · Full text

, , +13

X-Linked Thrombophilia with a Mutant Factor IX (Factor IX Padua)

Paolo Simioni, Daniela Tormene, Giulio Tognin et al. · New England Journal of Medicine · 2009 · 388 citations · Full text

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