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Engineering and selection of shuffled AAV genomes: A new strategy for producing targeted biological nanoparticles
Wuping Li et al.
MOLECULAR THERAPY (2008)
Small CRISPR RNAs guide antiviral defense in prokaryotes
Stan J. J. Brouns et al.
SCIENCE (2008)
Local dystrophin restoration with antisense oligonucleotide PRO051
Judith C. van Deutekom et al.
NEW ENGLAND JOURNAL OF MEDICINE (2007)
Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery
Angelo Lombardo et al.
NATURE BIOTECHNOLOGY (2007)
A bacterial effector acts as a plant transcription factor and induces a cell size regulator
Sabine Kay et al.
SCIENCE (2007)
Plant pathogen recognition mediated by promoter activation of the pepper Bs3 resistance gene
Patrick Roemer et al.
SCIENCE (2007)
The artificial 4-zinc-finger protein Bagly binds human utrophin promoter A at the endogenous chromosomal site and activates transcription
Annalisa Onori et al.
BIOCHEMISTRY AND CELL BIOLOGY-BIOCHIMIE ET BIOLOGIE CELLULAIRE (2007)
Hereditary myosin myopathies
Anders Oldfors
NEUROMUSCULAR DISORDERS (2007)
CD8+ T-cell responses to adeno-associated virus capsid in humans
Federico Mingozzi et al.
NATURE MEDICINE (2007)
CRISPR provides acquired resistance against viruses in prokaryotes
Rodolphe Barrangou et al.
SCIENCE (2007)
Non-viral gene therapy for Duchenne muscular dystrophy: Progress and challenges
Thomas A. Rando
BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE (2007)
Oculopharyngeal muscular dystrophy: Recent advances in the understanding of the molecular pathogenic mechanisms and treatment strategies
Aida Abu-Baker et al.
BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE (2007)
Homing endonuclease I-CreI derivatives with novel DNA target specificities
Laura E. Rosen et al.
NUCLEIC ACIDS RESEARCH (2006)
Molecular mechanisms of muscular dystrophies: old and new players
Kay E. Davies et al.
NATURE REVIEWS MOLECULAR CELL BIOLOGY (2006)
Facioscapulohumeral muscular dystrophy
Rabi Tawil et al.
MUSCLE & NERVE (2006)
Robust systemic transduction with AAV9 vectors in mice: Efficient global cardiac gene transfer superior to that of AAV8
Katsuya Inagaki et al.
MOLECULAR THERAPY (2006)
Computational redesign of endonuclease DNA binding and cleavage specificity
Justin Ashworth et al.
NATURE (2006)
Directed evolution and substrate specificity profile of homing endonuclease I-scel
JB Doyon et al.
JOURNAL OF THE AMERICAN CHEMICAL SOCIETY (2006)
Directed evolution of adeno-associated virus yields enhanced gene delivery vectors
N Maheshri et al.
NATURE BIOTECHNOLOGY (2006)
Engineering of large numbers of highly specific homing endonucleases that induce recombination on novel DNA targets
S Arnould et al.
JOURNAL OF MOLECULAR BIOLOGY (2006)
Trehalose reduces aggregate formation and delays pathology in a transgenic mouse model of oculopharyngeal muscular dystrophy
JE Davies et al.
HUMAN MOLECULAR GENETICS (2006)
Adeno-associated virus serotypes: Vector toolkit for human gene therapy
Zhijian Wu et al.
MOLECULAR THERAPY (2006)
Towards safe, non-viral therapeutic gene expression in humans
DJ Glover et al.
NATURE REVIEWS GENETICS (2005)
Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart
Z Wang et al.
NATURE BIOTECHNOLOGY (2005)
Adeno-associated virus-mediated microdystrophin expression protects young mdx muscle from contraction-induced injury
MJ Liu et al.
MOLECULAR THERAPY (2005)
Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping
A Goyenvalle et al.
SCIENCE (2004)
Safety and efficacy of factor IX gene transfer to skeletal muscle in murine and canine hemophilia B models by adeno-associated viral vector serotype 1
VR Arruda et al.
BLOOD (2004)
Integration of adeno-associated virus (AAV) and recombinant AAV vectors
DM McCarty et al.
ANNUAL REVIEW OF GENETICS (2004)
Site-selective in vivo targeting of cytosine-5 DNA methylation by zinc-finger proteins
CD Carvin et al.
NUCLEIC ACIDS RESEARCH (2003)
Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer
SE Raper et al.
MOLECULAR GENETICS AND METABOLISM (2003)
Chimeric recombinases with designed DNA sequence recognition
A Akopian et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2003)
Chimeric nucleases stimulate gene targeting in human cells
MH Porteus et al.
SCIENCE (2003)
Hypomethylation of D4Z4 in 4q-linked and non-4q-linked facioscapulohumeral muscular dystrophy
PGM van Overveld et al.
NATURE GENETICS (2003)
Non-viral gene delivery in skeletal muscle: a protein factory
QL Lu et al.
GENE THERAPY (2003)
Inappropriate gene activation in FSHD: A repressor complex binds a chromosomal repeat deleted in dystrophic muscle
D Gabellini et al.
CELL (2002)
Aggregate-prone proteins with polyglutamine and polyalanine expansions are degraded by autophagy
B Ravikumar et al.
HUMAN MOLECULAR GENETICS (2002)
Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model
B Wang et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2000)
Recombinant adenoviral vectors have adjuvant activity and stimulate T cell responses against tumor cells
SB Geutskens et al.
GENE THERAPY (2000)
The artificial zinc finger coding gene 'Jazz' binds the utrophin promoter and activates transcription
N Corbi et al.
GENE THERAPY (2000)
Positive and negative regulation of endogenous genes by designed transcription factors
RR Beerli et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2000)