Treatment of phenylketonuria using minicircle-based naked-DNA gene transfer to murine liver

Computationally designed liver-specific transcriptional modules and hyperactive factor IX improve hepatic gene therapy

(2014) N. Nair et al.   BLOOD

Minicircle DNA electrotransfer for efficient tissue-targeted gene delivery

(2012) S Chabot et al.   GENE THERAPY

Quantification of phenylalanine hydroxylase activity by isotope-dilution liquid chromatography–electrospray ionization tandem mass spectrometry

(2012) Caroline Heintz et al.   MOLECULAR GENETICS AND METABOLISM

The Extragenic Spacer Length Between the 5′ and 3′ Ends of the Transgene Expression Cassette Affects Transgene Silencing From Plasmid-based Vectors

(2012) Jiamiao Lu et al.   MOLECULAR THERAPY

The AAV Vector Toolkit: Poised at the Clinical Crossroads

(2012) Aravind Asokan et al.   MOLECULAR THERAPY

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

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

Complete restoration of phenylalanine oxidation in phenylketonuria mouse by a self-complementary adeno-associated virus vector

(2011) Hiroya Yagi et al.   JOURNAL OF GENE MEDICINE

Development of S/MAR minicircles for enhanced and persistent transgene expression in the mouse liver

(2011) Orestis Argyros et al.   JOURNAL OF MOLECULAR MEDICINE-JMM

Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges

(2011) Federico Mingozzi et al.   NATURE REVIEWS GENETICS

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

(2011) Mark A. Kay   NATURE REVIEWS GENETICS

Translating Sleeping Beauty transposition into cellular therapies: Victories and challenges

(2010) Zsuzsanna Izsvák et al.   BIOESSAYS

Transgene Loss and Changes in the Promoter Methylation Status as Determinants for Expression Duration in Nonviral Gene Transfer for Factor IX

(2010) Jörg Schüttrumpf et al.   HUMAN GENE THERAPY

Muscle Damage After Delivery of Naked Plasmid DNA into Skeletal Muscles Is Batch Dependent

(2010) Christine I. Wooddell et al.   HUMAN GENE THERAPY

Phenylketonuria

(2010) Nenad Blau et al.   LANCET

Minicircle DNA-based Gene Therapy Coupled With Immune Modulation Permits Long-term Expression of α-L-Iduronidase in Mice With Mucopolysaccharidosis Type I

(2010) Mark J Osborn et al.   MOLECULAR THERAPY

Conserved role of intragenic DNA methylation in regulating alternative promoters

(2010) Alika K. Maunakea et al.   NATURE

A robust system for production of minicircle DNA vectors

(2010) Mark A Kay et al.   NATURE BIOTECHNOLOGY

Progress and Prospects: The design and production of plasmid vectors

(2009) D R Gill et al.   GENE THERAPY

Comparison of Adeno-Associated Virus Pseudotype 1, 2, and 8 Vectors Administered by Intramuscular Injection in the Treatment of Murine Phenylketonuria

(2009) Alexandre Rebuffat et al.   HUMAN GENE THERAPY

Image-guided, Lobe-specific Hydrodynamic Gene Delivery to Swine Liver

(2009) Kenya Kamimura et al.   MOLECULAR THERAPY

Minimally invasive and selective hydrodynamic gene therapy of liver segments in the pig and human

(2008) S E Khorsandi et al.   CANCER GENE THERAPY

Episomal Vectors for Gene Therapy

(2008) Anja Ehrhardt et al.   CURRENT GENE THERAPY

Direct comparison of hepatocyte-specific expression cassettes following adenoviral and nonviral hydrodynamic gene transfer

(2008) F Jacobs et al.   GENE THERAPY

Persistent episomal transgene expression in liver following delivery of a scaffold/matrix attachment region containing non-viral vector

(2008) O Argyros et al.   GENE THERAPY

Minicircle-DNA production by site specific recombination and protein-DNA interaction chromatography

(2008) Peter Mayrhofer et al.   JOURNAL OF GENE MEDICINE

Correction of Murine PKU Following AAV-mediated Intramuscular Expression of a Complete Phenylalanine Hydroxylating System

(2008) Zhaobing Ding et al.   MOLECULAR THERAPY

Silencing of Episomal Transgene Expression in Liver by Plasmid Bacterial Backbone DNA Is Independent of CpG Methylation

(2008) Zhi-Ying Chen et al.   MOLECULAR THERAPY

CpG-free plasmids confer reduced inflammation and sustained pulmonary gene expression

(2008) Stephen C Hyde et al.   NATURE BIOTECHNOLOGY

Innate immune response induced by gene delivery vectors

(2007) Haruna Sakurai et al.   INTERNATIONAL JOURNAL OF PHARMACEUTICS