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

Hemophilia Gene Therapy: A Holy Grail Found

(2011) Katherine P Ponder   MOLECULAR THERAPY

Gene therapy finds its niche

(2011) Cormac Sheridan   NATURE BIOTECHNOLOGY

The expanding universe of transposon technologies for gene and cell engineering

(2011) Zoltán Ivics et al.   Mobile DNA

Inherited epidermolysis bullosa: past, present, and future

(2010) Jo-David Fine   Annals of the New York Academy of Sciences

Update on gene therapy for immunodeficiencies

(2010) Donald B. Kohn   CLINICAL IMMUNOLOGY

Production of Retroviral Vectors: Review

(2010) Ana S. Coroadinha et al.   CURRENT GENE THERAPY

GM-CSF-armed, replication-competent viruses for cancer

(2010) James M. Burke   CYTOKINE & GROWTH FACTOR REVIEWS

Gene Therapy for Recessive Dystrophic Epidermolysis Bullosa

(2010) Matthias Titeux et al.   DERMATOLOGIC CLINICS

Gene therapy for muscle disease

(2010) Yuko Miyagoe-Suzuki et al.   EXPERIMENTAL CELL RESEARCH

Progress and prospects: nuclear import of nonviral vectors

(2010) A P Lam et al.   GENE THERAPY

Prevention of spontaneous bleeding in dogs with haemophilia A and haemophilia B

(2010) T. C. NICHOLS et al.   HAEMOPHILIA

Prevalence of Serum IgG and Neutralizing Factors Against Adeno-Associated Virus (AAV) Types 1, 2, 5, 6, 8, and 9 in the Healthy Population: Implications for Gene Therapy Using AAV Vectors

(2010) Sylvie Boutin et al.   HUMAN GENE THERAPY

Argonaute proteins are key determinants of RNAi efficacy, toxicity, and persistence in the adult mouse liver

(2010) Dirk Grimm et al.   JOURNAL OF CLINICAL INVESTIGATION

Biodistribution and safety assessment of AAV2-GAD following intrasubthalamic injection in the rat

(2010) Helen L. Fitzsimons et al.   JOURNAL OF GENE MEDICINE

Minireview: Switching on Progesterone Receptor Expression with Duplex RNA

(2010) Bethany A. Janowski et al.   MOLECULAR ENDOCRINOLOGY

Treatment of Cancer Patients With a Serotype 5/3 Chimeric Oncolytic Adenovirus Expressing GMCSF

(2010) Anniina Koski et al.   MOLECULAR THERAPY

Eight Years of Clinical Improvement in MPTP-Lesioned Primates After Gene Therapy With AAV2-hAADC

(2010) Piotr Hadaczek et al.   MOLECULAR THERAPY

SERCA2a Gene Transfer Enhances eNOS Expression and Activity in Endothelial Cells

(2010) Lahouaria Hadri et al.   MOLECULAR THERAPY

Hyperactive Sleeping Beauty Transposase Enables Persistent Phenotypic Correction in Mice and a Canine Model for Hemophilia B

(2010) Martin A Hausl et al.   MOLECULAR THERAPY

A Phase I Study of Aromatic L-Amino Acid Decarboxylase Gene Therapy for Parkinson's Disease

(2010) Shin-ichi Muramatsu et al.   MOLECULAR THERAPY

The Status of Exon Skipping as a Therapeutic Approach to Duchenne Muscular Dystrophy

(2010) Qi-Long Lu et al.   MOLECULAR THERAPY

Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

(2010) Marina Cavazzana-Calvo et al.   NATURE

A robust system for production of minicircle DNA vectors

(2010) Mark A Kay et al.   NATURE BIOTECHNOLOGY

Genome editing with engineered zinc finger nucleases

(2010) Fyodor D. Urnov et al.   NATURE REVIEWS GENETICS

Stem-Cell Gene Therapy for the Wiskott–Aldrich Syndrome

(2010) Kaan Boztug et al.   NEW ENGLAND JOURNAL OF MEDICINE

RNA-Based Gene Therapy for HIV with Lentiviral Vector-Modified CD34+ Cells in Patients Undergoing Transplantation for AIDS-Related Lymphoma

(2010) D. L. DiGiusto et al.   Science Translational Medicine

Novel adenovirus vector-based vaccines for HIV-1

(2010) Dan H Barouch   Current Opinion in HIV and AIDS

Overview of STEP and Phambili trial results: two phase IIb test-of-concept studies investigating the efficacy of MRK adenovirus type 5 gag/pol/nef subtype B HIV vaccine

(2010) Glenda Gray et al.   Current Opinion in HIV and AIDS

Chemokine receptor 5 knockout strategies

(2010) Paula Cannon et al.   Current Opinion in HIV and AIDS

Nonviral Gene Delivery: Principle, Limitations, and Recent Progress

(2009) Mohammed S. Al-Dosari et al.   AAPS Journal

Gene therapy with human and mouse T-cell receptors mediates cancer regression and targets normal tissues expressing cognate antigen

(2009) L. A. Johnson et al.   BLOOD

AAV-1-mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells

(2009) F. Mingozzi et al.   BLOOD

Therapeutic microRNA Delivery Suppresses Tumorigenesis in a Murine Liver Cancer Model

(2009) Janaiah Kota et al.   CELL

Progress Towards Liver and Lung-Directed Gene Therapy with Helper- Dependent Adenoviral Vectors

(2009) Nicola Brunetti-Pierri et al.   CURRENT GENE THERAPY

Non-viral vectors in cystic fibrosis gene therapy: recent developments and future prospects

(2009) IA Pringle et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

Foamy virus vectors for gene transfer

(2009) Grant D Trobridge   EXPERT OPINION ON BIOLOGICAL THERAPY

Progress and Prospects: The design and production of plasmid vectors

(2009) D R Gill et al.   GENE THERAPY

The influence of innate and pre-existing immunity on adenovirus therapy

(2009) Anne K. Zaiss et al.   JOURNAL OF CELLULAR BIOCHEMISTRY

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

Improved Retinal Function in a Mouse Model of Dominant Retinitis Pigmentosa Following AAV-delivered Gene Therapy

(2009) Naomi Chadderton et al.   MOLECULAR THERAPY

Enhanced Exon-skipping Induced by U7 snRNA Carrying a Splicing Silencer Sequence: Promising Tool for DMD Therapy

(2009) Aurélie Goyenvalle et al.   MOLECULAR THERAPY

New Insights on Adenovirus as Vaccine Vectors

(2009) Marcio O Lasaro et al.   MOLECULAR THERAPY

Safety and tolerability of putaminal AADC gene therapy for Parkinson disease

(2009) C. W. Christine et al.   NEUROLOGY

Gefitinib or Carboplatin–Paclitaxel in Pulmonary Adenocarcinoma

(2009) Tony S. Mok et al.   NEW ENGLAND JOURNAL OF MEDICINE

Efficient gene therapy-based method for the delivery of therapeutics to primate cortex

(2009) A. P. Kells et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy

(2009) M. L. Brantly et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy

(2009) N. Cartier et al.   SCIENCE

Development of gene therapy for blood disorders

(2008) A. W. Nienhuis   BLOOD

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

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

Gene Therapy Using Adeno-Associated Virus Vectors

(2008) S. Daya et al.   CLINICAL MICROBIOLOGY REVIEWS

Clinical gene therapy using recombinant adeno-associated virus vectors

(2008) C Mueller et al.   GENE THERAPY

Design of a Phase 1/2 Trial of Intracoronary Administration of AAV1/SERCA2a in Patients With Heart Failure

(2008) Roger J. Hajjar et al.   JOURNAL OF CARDIAC FAILURE

Frequency and Spectrum of Genomic Integration of Recombinant Adeno-Associated Virus Serotype 8 Vector in Neonatal Mouse Liver

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

In Vitro and In Vivo Gene Therapy Vector Evolution via Multispecies Interbreeding and Retargeting of Adeno-Associated Viruses

(2008) D. Grimm et al.   JOURNAL OF VIROLOGY

The Host Response to Adenovirus, Helper-dependent Adenovirus, and Adeno-associated Virus in Mouse Liver

(2008) Anton P McCaffrey et al.   MOLECULAR THERAPY

Engineering and Selection of Shuffled AAV Genomes: A New Strategy for Producing Targeted Biological Nanoparticles

(2008) Wuping Li et al.   MOLECULAR THERAPY

AAV Vector–mediated RNAi of Mutant Huntingtin Expression Is Neuroprotective in a Novel Genetic Rat Model of Huntington's Disease

(2008) Nicholas R Franich 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

In vivo reprogramming of adult pancreatic exocrine cells to β-cells

(2008) Qiao Zhou et al.   NATURE

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

(2008) Stephen C Hyde et al.   NATURE BIOTECHNOLOGY

Virus-specific T cells engineered to coexpress tumor-specific receptors: persistence and antitumor activity in individuals with neuroblastoma

(2008) Martin A Pule et al.   NATURE MEDICINE

Real-time MR imaging of adeno-associated viral vector delivery to the primate brain

(2008) Massimo S. Fiandaca et al.   NEUROIMAGE

Results from a phase I safety trial of hAADC gene therapy for Parkinson disease

(2008) J. L. Eberling et al.   NEUROLOGY

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

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

Next generation of adeno-associated virus 2 vectors: Point mutations in tyrosines lead to high-efficiency transduction at lower doses

(2008) L. Zhong et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA