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Article

Evaluation of engineered AAV capsids for hepatic factor IX gene transfer in murine and canine models.

Journal of translational medicine - 1 May 2017

Markusic David M, Nichols Timothy C, Merricks Elizabeth P, Palaschak Brett, Zolotukhin Irene, Marsic Damien, Zolotukhin Sergei, Srivastava Arun, Herzog Roland W

Abstract excerpt

BACKGROUND: Adeno-associated virus (AAV) gene therapy vectors have shown the best outcomes in human clinical studies for the treatment of genetic diseases such as hemophilia. However, these pivotal investigations have also identified several challenges. For example, high vector doses are often used for hepatic gene transfer, and cytotoxic T lymphocyte responses against viral capsid may occur. Therefore, achieving...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Source

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Identifiers

PMID: 28460646
DOI: 10.1186/s12967-017-1200-1
PMCID: PMC5412045

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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