Zinc-finger nuclease-mediated gene correction using single AAV vector transduction and enhancement by Food and Drug Administration-approved drugs

In vivo genome editing restores haemostasis in a mouse model of haemophilia

(2011) Hojun Li et al.   NATURE

Gene therapy for ocular diseases

(2010) M. M. Liu et al.   BRITISH JOURNAL OF OPHTHALMOLOGY

Cationic lipid-mediated nucleic acid delivery: beyond being cationic

(2010) N. Madhusudhana Rao   CHEMISTRY AND PHYSICS OF LIPIDS

Self-complementary AAV mediates gene targeting and enhances endonuclease delivery for double-strand break repair

(2010) M L Hirsch et al.   GENE THERAPY

Targeting DNA Double-Strand Breaks with TAL Effector Nucleases

(2010) M. Christian et al.   GENETICS

Functional genomics, proteomics, and regulatory DNA analysis in isogenic settings using zinc finger nuclease-driven transgenesis into a safe harbor locus in the human genome

(2010) R. C. DeKelver et al.   GENOME RESEARCH

Gene Therapy for Adenosine Deaminase Deficiency

(2010) Barbara Cappelli et al.   IMMUNOLOGY AND ALLERGY CLINICS OF NORTH AMERICA

Gene Correction by Homologous Recombination With Zinc Finger Nucleases in Primary Cells From a Mouse Model of a Generic Recessive Genetic Disease

(2010) Jon P Connelly et al.   MOLECULAR THERAPY

Proteasome Inhibitors Enhance Gene Delivery by AAV Virus Vectors Expressing Large Genomes in Hemophilia Mouse and Dog Models: A Strategy for Broad Clinical Application

(2010) Paul E Monahan 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

Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo

(2010) Nathalia Holt et al.   NATURE BIOTECHNOLOGY

A TALE nuclease architecture for efficient genome editing

(2010) Jeffrey C Miller et al.   NATURE BIOTECHNOLOGY

20 years of gene therapy for SCID

(2010) Alain Fischer et al.   NATURE IMMUNOLOGY

Zinc-finger nuclease-driven targeted integration into mammalian genomes using donors with limited chromosomal homology

(2010) Salvatore J. Orlando et al.   NUCLEIC ACIDS RESEARCH

Establishment of an AAV Reverse Infection-Based Array

(2010) Xiaoyan Dong et al.   PLoS One

Progress and prospects: gene therapy for inherited immunodeficiencies

(2009) W Qasim et al.   GENE THERAPY

Gene therapy with a promoter targeting both rods and cones rescues retinal degeneration caused by AIPL1 mutations

(2009) X Sun et al.   GENE THERAPY

Fate of Recombinant Adeno-Associated Viral Vector Genomes During DNA Double-Strand Break-Induced Gene Targeting in Human Cells

(2009) Katharina Gellhaus et al.   HUMAN GENE THERAPY

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

Proteasome Inhibitors Decrease AAV2 Capsid derived Peptide Epitope Presentation on MHC Class I Following Transduction

(2009) Jonathan D Finn et al.   MOLECULAR THERAPY

Gene Therapy for Leber's Congenital Amaurosis is Safe and Effective Through 1.5 Years After Vector Administration

(2009) Francesca Simonelli et al.   MOLECULAR THERAPY

Attenuation of Zinc Finger Nuclease Toxicity by Small-Molecule Regulation of Protein Levels

(2009) Shondra M. Pruett-Miller et al.   PLoS Genetics

Development of optimal bicistronic lentiviral vectors facilitates high-level TCR gene expression and robust tumor cell recognition

(2008) S Yang et al.   GENE THERAPY

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

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

Comparison of Zinc Finger Nucleases for Use in Gene Targeting in Mammalian Cells

(2008) Shondra M Pruett-Miller et al.   MOLECULAR THERAPY

Zinc-finger Nucleases: The Next Generation Emerges

(2008) Toni Cathomen et al.   MOLECULAR THERAPY

Analysis of AAV Serotypes 1–9 Mediated Gene Expression and Tropism in Mice After Systemic Injection

(2008) Carmela Zincarelli et al.   MOLECULAR THERAPY

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