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Article

Correction of murine hemophilia A and immunological differences of factor VIII variants delivered by helper-dependent adenoviral vectors.

Molecular therapy : the journal of the American Society of Gene Therapy - 1 Dec 2007

Cerullo Vincenzo, Seiler Michael P, Mane Viraj, Cela Racel, Clarke Christian, Kaufman Randal J, Pipe Steven W, Lee Brendan

Abstract excerpt

Bioengineering of the factor VIII (FVIII) molecule has led to the production of variants that overcome poor secretion and/or rapid inactivation. We tested six modified FVIII variants for in vivo efficacy by expressing them from helper-dependent adenoviral (HD-Ad) vectors. We constructed a wild-type (WT) variant, a B-domain-deleted (BDD) variant, a point mutant for improved secretion (F309S), a variant with a...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 17848960
DOI: 10.1038/sj.mt.6300308

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