Adeno-associated virus: a key to the human genome?

Near-perfect infectivity of wild-type AAV as benchmark for infectivity of recombinant AAV vectors

(2010) N Zeltner et al.   GENE THERAPY

Progress toward the clinical application of patient-specific pluripotent stem cells

(2010) Evangelos Kiskinis et al.   JOURNAL OF CLINICAL INVESTIGATION

Frequent Endonuclease Cleavage at Off-target Locations In Vivo

(2010) Lisa M Petek et al.   MOLECULAR THERAPY

Efficient KRT14 Targeting and Functional Characterization of Transplanted Human Keratinocytes for the Treatment of Epidermolysis Bullosa Simplex

(2010) Lisa M Petek et al.   MOLECULAR THERAPY

Engineering of Human Pluripotent Stem Cells by AAV-mediated Gene Targeting

(2010) Iram F Khan et al.   MOLECULAR THERAPY

BAAV Transcytosis Requires an Interaction with β-1-4 Linked- Glucosamine and gp96

(2010) Giovanni Di Pasquale et al.   PLoS One

A viral assembly factor promotes AAV2 capsid formation in the nucleolus

(2010) F. Sonntag et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Adenovirus–Adeno-Associated Virus Hybrid for Large-Scale Recombinant Adeno-Associated Virus Production

(2009) Hongwei Zhang et al.   HUMAN GENE THERAPY

Human RPE65 Gene Therapy for Leber Congenital Amaurosis: Persistence of Early Visual Improvements and Safety at 1 Year

(2009) Artur V. Cideciyan et al.   HUMAN GENE THERAPY

Transient Transfection Methods for Clinical Adeno-Associated Viral Vector Production

(2009) J. Fraser Wright   HUMAN GENE THERAPY

Producing Recombinant Adeno-Associated Virus in Foster Cells: Overcoming Production Limitations Using a Baculovirus–Insect Cell Expression Strategy

(2009) Tamas Virag et al.   HUMAN GENE THERAPY

Large-Scale Adeno-Associated Viral Vector Production Using a Herpesvirus-Based System Enables Manufacturing for Clinical Studies

(2009) Nathalie Clément et al.   HUMAN GENE THERAPY

The TLR9-MyD88 pathway is critical for adaptive immune responses to adeno-associated virus gene therapy vectors in mice

(2009) Jiangao Zhu et al.   JOURNAL OF CLINICAL INVESTIGATION

Worldwide Epidemiology of Neutralizing Antibodies to Adeno‐Associated Viruses

(2009) Roberto Calcedo et al.   JOURNAL OF INFECTIOUS DISEASES

Adeno-Associated Virus Small Rep Proteins Are Modified with at Least Two Types of Polyubiquitination

(2009) K. D. Farris et al.   JOURNAL OF VIROLOGY

Transcriptional Analysis of the Adeno-Associated Virus Integration Site

(2009) N. Dutheil et al.   JOURNAL OF VIROLOGY

Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial

(2009) Albert M Maguire et al.   LANCET

Reengineering a receptor footprint of adeno-associated virus enables selective and systemic gene transfer to muscle

(2009) Aravind Asokan et al.   NATURE BIOTECHNOLOGY

Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases

(2009) Dirk Hockemeyer et al.   NATURE BIOTECHNOLOGY

Efficient targeting of a SCID gene by an engineered single-chain homing endonuclease

(2009) Sylvestre Grizot et al.   NUCLEIC ACIDS RESEARCH

Site-specific integration of adeno-associated virus involves partial duplication of the target locus

(2009) E. Henckaerts et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

DNA Structure Modulates the Oligomerization Properties of the AAV Initiator Protein Rep68

(2009) Jorge Mansilla-Soto et al.   PLoS Pathogens

Adeno-associated virus infection and cervical neoplasia: is there a protective role against human papillomavirus-related carcinogenesis?

(2008) Theodoros Agorastos et al.   EUROPEAN JOURNAL OF CANCER PREVENTION

AAV-mediated gene transfer for the treatment of hemophilia B: problems and prospects

(2008) N C Hasbrouck et al.   GENE THERAPY

Clinical gene therapy using recombinant adeno-associated virus vectors

(2008) C Mueller et al.   GENE THERAPY

Strategies for improving the transduction efficiency of single-stranded adeno-associated virus vectors in vitro and in vivo

(2008) G R Jayandharan et al.   GENE THERAPY

Tailoring the AAV vector capsid for gene therapy

(2008) L H Vandenberghe et al.   GENE THERAPY

Treatment of Leber Congenital Amaurosis Due to RPE65 Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a Phase I Trial

(2008) William W. Hauswirth et al.   HUMAN GENE THERAPY

Identification of Cellular Proteins That Interact with the Adeno-Associated Virus Rep Protein

(2008) K. Nash et al.   JOURNAL OF VIROLOGY

Enhancement of Adeno-Associated Virus Infection by Mobilizing Capsids into and Out of the Nucleolus

(2008) J. S. Johnson et al.   JOURNAL OF VIROLOGY

E4Orf6-E1B-55k-Dependent Degradation of De Novo-Generated Adeno-Associated Virus Type 5 Rep52 and Capsid Proteins Employs a Cullin 5-Containing E3 Ligase Complex

(2008) R. Nayak et al.   JOURNAL OF VIROLOGY

Zinc-finger Nucleases: The Next Generation Emerges

(2008) Toni Cathomen et al.   MOLECULAR THERAPY

Self-complementary AAV Vectors; Advances and Applications

(2008) Douglas M McCarty   MOLECULAR THERAPY

Mutational Derivatives of PhiC31 Integrase With Increased Efficiency and Specificity

(2008) Annahita Keravala et al.   MOLECULAR THERAPY

Molecular basis of xeroderma pigmentosum group C DNA recognition by engineered meganucleases

(2008) Pilar Redondo et al.   NATURE

Human cardiovascular progenitor cells develop from a KDR+ embryonic-stem-cell-derived population

(2008) Lei Yang et al.   NATURE

Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases

(2008) Elena E Perez et al.   NATURE BIOTECHNOLOGY

Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis

(2008) Albert M. Maguire et al.   NEW ENGLAND JOURNAL OF MEDICINE

Effect of Gene Therapy on Visual Function in Leber's Congenital Amaurosis

(2008) James W.B. Bainbridge et al.   NEW ENGLAND JOURNAL OF MEDICINE

Leber congenital amaurosis: Genes, proteins and disease mechanisms

(2008) Anneke I. den Hollander et al.   PROGRESS IN RETINAL AND EYE RESEARCH

Single-polarity Recombinant Adeno-associated Virus 2 Vector-mediated Transgene Expression In Vitro and In Vivo: Mechanism of Transduction

(2007) Li Zhong et al.   MOLECULAR THERAPY

Gene Targeting of Mutant COL1A2 Alleles in Mesenchymal Stem Cells From Individuals With Osteogenesis Imperfecta

(2007) Joel R Chamberlain et al.   MOLECULAR THERAPY