Ultrasound-targeted hepatic delivery of factor IX in hemophiliac mice

Promoterless gene targeting without nucleases ameliorates haemophilia B in mice

(2014) A. Barzel et al.   NATURE

Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

(2014) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Ultrasound Directs a Transposase System for Durable Hepatic Gene Delivery in Mice

(2013) Cynthia D. Anderson et al.   ULTRASOUND IN MEDICINE AND BIOLOGY

Minicircle DNA Vectors Achieve Sustained Expression Reflected by Active Chromatin and Transcriptional Level

(2012) Lia E. Gracey Maniar et al.   MOLECULAR THERAPY

Repeated and targeted transfer of angiogenic plasmids into the infarcted rat heart via ultrasound targeted microbubble destruction enhances cardiac repair

(2011) Hiroko Fujii et al.   EUROPEAN HEART JOURNAL

Long-term phenotypic correction in factor IX knockout mice by using phiC31 integrase-mediated gene therapy

(2011) A Keravala et al.   GENE THERAPY

State-of-the-art gene-based therapies: the road ahead

(2011) Mark A. Kay   NATURE REVIEWS GENETICS

Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B

(2011) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Helper-independent piggyBac plasmids for gene delivery approaches: Strategies for avoiding potential genotoxic effects

(2010) J. Urschitz et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Effect of nuclear localization and hydrodynamic delivery-induced cell division on φC31 integrase activity

(2009) L E Woodard et al.   GENE THERAPY

Molecular evolution of a novel hyperactive Sleeping Beauty transposase enables robust stable gene transfer in vertebrates

(2009) Lajos Mátés et al.   NATURE GENETICS

Mutational Derivatives of PhiC31 Integrase With Increased Efficiency and Specificity

(2008) Annahita Keravala et al.   MOLECULAR THERAPY