Efficient modification of CCR5 in primary human hematopoietic cells using a megaTAL nuclease and AAV donor template

Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells

(2015) M. D. Hoban et al.   BLOOD

Intraosseous Delivery of Lentiviral Vectors Targeting Factor VIII Expression in Platelets Corrects Murine Hemophilia A

(2015) Xuefeng Wang et al.   MOLECULAR THERAPY

Adoptive cellular therapy: A race to the finish line

(2015) Carl H. June et al.   Science Translational Medicine

A highly compact epitope-based marker/suicide gene for easier and safer T-cell therapy

(2014) B. Philip et al.   BLOOD

Efficient Ablation of Genes in Human Hematopoietic Stem and Effector Cells using CRISPR/Cas9

(2014) Pankaj K. Mandal et al.   Cell Stem Cell

TALEN-mediated editing of endogenous T-cell receptors facilitates efficient reprogramming of T lymphocytes by lentiviral gene transfer

(2014) B Berdien et al.   GENE THERAPY

Going viral: chimeric antigen receptor T-cell therapy for hematological malignancies

(2014) Saar Gill et al.   IMMUNOLOGICAL REVIEWS

Gene Transfer Properties and Structural Modeling of Human Stem Cell-derived AAV

(2014) Laura J Smith et al.   MOLECULAR THERAPY

Targeted genome editing in human repopulating haematopoietic stem cells

(2014) Pietro Genovese et al.   NATURE

Gene Editing of CCR5 in Autologous CD4 T Cells of Persons Infected with HIV

(2014) Pablo Tebas et al.   NEW ENGLAND JOURNAL OF MEDICINE

Progressive engineering of a homing endonuclease genome editing reagent for the murine X-linked immunodeficiency locus

(2014) Yupeng Wang et al.   NUCLEIC ACIDS RESEARCH

Easy quantitative assessment of genome editing by sequence trace decomposition

(2014) Eva K. Brinkman et al.   NUCLEIC ACIDS RESEARCH

Optimizing the transduction efficiency of capsid-modified AAV6 serotype vectors in primary human hematopoietic stem cells in vitro and in a xenograft mouse model in vivo

(2013) Liujiang Song et al.   CYTOTHERAPY

Generation of an HIV Resistant T-cell Line by Targeted “Stacking” of Restriction Factors

(2013) Richard A Voit et al.   MOLECULAR THERAPY

megaTALs: a rare-cleaving nuclease architecture for therapeutic genome engineering

(2013) Sandrine Boissel et al.   NUCLEIC ACIDS RESEARCH

High-Efficiency Transduction of Primary Human Hematopoietic Stem Cells and Erythroid Lineage-Restricted Expression by Optimized AAV6 Serotype Vectors In Vitro and in a Murine Xenograft Model In Vivo

(2013) Liujiang Song et al.   PLoS One

A survey of ex vivo/in vitro transduction efficiency of mammalian primary cells and cell lines with Nine natural adeno-associated virus (AAV1-9) and one engineered adeno-associated virus serotype

(2013) Brian L Ellis et al.   Virology Journal

Comparison of Insulators and Promoters for Expression of the Wiskott–Aldrich Syndrome Protein Using Lentiviral Vectors

(2013) Rachel M. Koldej et al.   Human Gene Therapy Clinical Development

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

(2012) B L Ellis et al.   GENE THERAPY

Universal Real-Time PCR for the Detection and Quantification of Adeno-Associated Virus Serotype 2-Derived Inverted Terminal Repeat Sequences

(2012) Christine Aurnhammer et al.   Human Gene Therapy Methods

Generation of CD19-chimeric antigen receptor modified CD8+ T cells derived from virus-specific central memory T cells

(2011) S. Terakura et al.   BLOOD

Versatile and Efficient Genome Editing in Human Cells by Combining Zinc-Finger Nucleases With Adeno-Associated Viral Vectors

(2011) Eva-Maria Händel et al.   HUMAN GENE THERAPY

Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector

(2011) Dawn E Bowles et al.   MOLECULAR THERAPY

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

(2011) Hojun Li et al.   NATURE

Broad neutralization coverage of HIV by multiple highly potent antibodies

(2011) Laura M. Walker et al.   NATURE

Tracking genome engineering outcome at individual DNA breakpoints

(2011) Michael T Certo et al.   NATURE METHODS

AAV-mediated gene targeting methods for human cells

(2011) Iram F Khan et al.   Nature Protocols

Efficient design and assembly of custom TALEN and other TAL effector-based constructs for DNA targeting

(2011) Tomas Cermak et al.   NUCLEIC ACIDS RESEARCH

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

(2010) M L Hirsch et al.   GENE THERAPY

A TALE nuclease architecture for efficient genome editing

(2010) Jeffrey C Miller et al.   NATURE BIOTECHNOLOGY

Aryl Hydrocarbon Receptor Antagonists Promote the Expansion of Human Hematopoietic Stem Cells

(2010) Anthony E. Boitano et al.   SCIENCE

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

Chimeric Receptors Containing CD137 Signal Transduction Domains Mediate Enhanced Survival of T Cells and Increased Antileukemic Efficacy In Vivo

(2009) Michael C. Milone et al.   MOLECULAR THERAPY

High-resolution profiling of homing endonuclease binding and catalytic specificity using yeast surface display

(2009) Jordan Jarjour et al.   NUCLEIC ACIDS RESEARCH

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

(2009) N. Cartier et al.   SCIENCE

Development of a simple and highly sensitive mutation screening system by enzyme mismatch cleavage with optimized conditions for standard laboratories

(2008) Takanori Tsuji et al.   ELECTROPHORESIS

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

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

(2008) Elena E Perez et al.   NATURE BIOTECHNOLOGY