Promoterless gene targeting without nucleases ameliorates haemophilia B in mice View Full Text


Ontology type: schema:ScholarlyArticle      Open Access: True


Article Info

DATE

2014-10-29

AUTHORS

A. Barzel, N. K. Paulk, Y. Shi, Y. Huang, K. Chu, F. Zhang, P. N. Valdmanis, L. P. Spector, M. H. Porteus, K. M. Gaensler, M. A. Kay

ABSTRACT

Promoterless recombinant adeno-associated virus is used without nucleases to target the human coagulation factor IX gene to the liver-expressed albumin locus in haemophilia B mice, with an on-target integration into ∼0.5% of the albumin alleles in hepatocytes; stable F9 plasma levels at 7–20% of normal were obtained, leading to normal coagulation times in treated factor-IX-deficient mice. More... »

PAGES

360-364

Journal

TITLE

Nature

ISSUE

7534

VOLUME

517

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  • Identifiers

    URI

    http://scigraph.springernature.com/pub.10.1038/nature13864

    DOI

    http://dx.doi.org/10.1038/nature13864

    DIMENSIONS

    https://app.dimensions.ai/details/publication/pub.1039253377

    PUBMED

    https://www.ncbi.nlm.nih.gov/pubmed/25363772


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