Hematopoietic stem cell engineering at a crossroads

3'UTR-truncated Hmga2 cDNA causes MPN-like hematopoiesis by conferring a clonal growth advantage at the level of HSC in mice

(2011) K. Ikeda et al.   BLOOD

Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection

(2011) A. Biffi et al.   BLOOD

Dynamic Changes in the Copy Number of Pluripotency and Cell Proliferation Genes in Human ESCs and iPSCs during Reprogramming and Time in Culture

(2011) Louise C. Laurent et al.   Cell Stem Cell

The Use of Chromatin Insulators to Improve the Expression and Safety of Integrating Gene Transfer Vectors

(2011) David W. Emery   HUMAN GENE THERAPY

Contributions of Gene Marking to Cell and Gene Therapies

(2011) Cecilia N. Barese et al.   HUMAN GENE THERAPY

Insertion Sites in Engrafted Cells Cluster Within a Limited Repertoire of Genomic Areas After Gammaretroviral Vector Gene Therapy

(2011) Annette Deichmann et al.   MOLECULAR THERAPY

Somatic coding mutations in human induced pluripotent stem cells

(2011) Athurva Gore et al.   NATURE

Copy number variation and selection during reprogramming to pluripotency

(2011) Samer M. Hussein et al.   NATURE

Immunogenicity of induced pluripotent stem cells

(2011) Tongbiao Zhao et al.   NATURE

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

(2011) Hojun Li et al.   NATURE

TALEs of genome targeting

(2011) Jens Boch   NATURE BIOTECHNOLOGY

Tracking single hematopoietic stem cells in vivo using high-throughput sequencing in conjunction with viral genetic barcoding

(2011) Rong Lu et al.   NATURE BIOTECHNOLOGY

An unbiased genome-wide analysis of zinc-finger nuclease specificity

(2011) Richard Gabriel et al.   NATURE BIOTECHNOLOGY

Revealing off-target cleavage specificities of zinc-finger nucleases by in vitro selection

(2011) Vikram Pattanayak et al.   NATURE METHODS

Eliminating Cells Gone Astray

(2011) Michel Sadelain   NEW ENGLAND JOURNAL OF MEDICINE

Inducible Apoptosis as a Safety Switch for Adoptive Cell Therapy

(2011) Antonio Di Stasi et al.   NEW ENGLAND JOURNAL OF MEDICINE

A method to sequence and quantify DNA integration for monitoring outcome in gene therapy

(2011) Troy Brady et al.   NUCLEIC ACIDS RESEARCH

Genetic correction and analysis of induced pluripotent stem cells from a patient with gyrate atrophy

(2011) S. E. Howden et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Long-Term Persistence of a Polyclonal T Cell Repertoire After Gene Therapy for X-Linked Severe Combined Immunodeficiency

(2011) H. B. Gaspar et al.   Science Translational Medicine

Hematopoietic Stem Cell Gene Therapy for Adenosine Deaminase-Deficient Severe Combined Immunodeficiency Leads to Long-Term Immunological Recovery and Metabolic Correction

(2011) H. B. Gaspar et al.   Science Translational Medicine

The challenge of obtaining therapeutic levels of genetically modified hematopoietic stem cells in β-thalassemia patients

(2010) Derek A. Persons   Annals of the New York Academy of Sciences

Identification and Classification of Chromosomal Aberrations in Human Induced Pluripotent Stem Cells

(2010) Yoav Mayshar et al.   Cell Stem Cell

Modeling Disease in Human ESCs Using an Efficient BAC-Based Homologous Recombination System

(2010) Hoseok Song et al.   Cell Stem Cell

Highly Efficient Reprogramming to Pluripotency and Directed Differentiation of Human Cells with Synthetic Modified mRNA

(2010) Luigi Warren et al.   Cell Stem Cell

Update on gene therapy for immunodeficiencies

(2010) Donald B. Kohn   CLINICAL IMMUNOLOGY

Progress and prospects: Foamy virus vectors enter a new age

(2010) O Erlwein et al.   GENE THERAPY

TK.007: A Novel, Codon-Optimized HSVtk(A168H) Mutant for Suicide Gene Therapy

(2010) Ellen Preuß et al.   HUMAN GENE THERAPY

Lentivirus-mediated Reprogramming of Somatic Cells in the Absence of Transgenic Transcription Factors

(2010) Nicole M Kane et al.   MOLECULAR THERAPY

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

(2010) Iram F Khan et al.   MOLECULAR THERAPY

Zinc-finger Nuclease-induced Gene Repair With Oligodeoxynucleotides: Wanted and Unwanted Target Locus Modifications

(2010) Sarah Radecke et al.   MOLECULAR THERAPY

Nanoparticles Deliver Triplex-forming PNAs for Site-specific Genomic Recombination in CD34+ Human Hematopoietic Progenitors

(2010) Nicole A McNeer et al.   MOLECULAR THERAPY

Direct conversion of human fibroblasts to multilineage blood progenitors

(2010) Eva Szabo et al.   NATURE

In vivo imaging of haematopoietic cells emerging from the mouse aortic endothelium

(2010) Jean-Charles Boisset et al.   NATURE

Blood stem cells emerge from aortic endothelium by a novel type of cell transition

(2010) Karima Kissa et al.   NATURE

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

(2010) Marina Cavazzana-Calvo et al.   NATURE

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

Genomic safe harbors permit high β-globin transgene expression in thalassemia induced pluripotent stem cells

(2010) Eirini P Papapetrou et al.   NATURE BIOTECHNOLOGY

Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease

(2010) Stefan Stein et al.   NATURE MEDICINE

Stem-Cell Gene Therapy for the Wiskott–Aldrich Syndrome

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

The Lmo2 Oncogene Initiates Leukemia in Mice by Inducing Thymocyte Self-Renewal

(2010) M. P. McCormack et al.   SCIENCE

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

The genomic risk of somatic gene therapy

(2010) Olga Kustikova et al.   SEMINARS IN CANCER BIOLOGY

Sustained high-level polyclonal hematopoietic marking and transgene expression 4 years after autologous transplantation of rhesus macaques with SIV lentiviral vector-transduced CD34+ cells

(2009) Y.-J. Kim et al.   BLOOD

Gene Targeting of a Disease-Related Gene in Human Induced Pluripotent Stem and Embryonic Stem Cells

(2009) Jizhong Zou et al.   Cell Stem Cell

Progress and prospects: gene therapy for inherited immunodeficiencies

(2009) W Qasim et al.   GENE THERAPY

The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy

(2009) Eugenio Montini et al.   JOURNAL OF CLINICAL INVESTIGATION

Optimized Lentiviral Vector Design Improves Titer and Transgene Expression of Vectors Containing the Chicken β-Globin Locus HS4 Insulator Element

(2009) Hideki Hanawa et al.   MOLECULAR THERAPY

Mechanism of Reduction in Titers From Lentivirus Vectors Carrying Large Inserts in the 3′LTR

(2009) Fabrizia Urbinati et al.   MOLECULAR THERAPY

Insertional Transformation of Hematopoietic Cells by Self-inactivating Lentiviral and Gammaretroviral Vectors

(2009) Ute Modlich et al.   MOLECULAR THERAPY

Genomic and Functional Assays Demonstrate Reduced Gammaretroviral Vector Genotoxicity Associated With Use of the cHS4 Chromatin Insulator

(2009) Chang Long Li et al.   MOLECULAR THERAPY

Finding the needle in the hay stack: Hematopoietic stem cells in Fanconi anemia

(2009) Lars U.W. Müller et al.   MUTATION RESEARCH-FUNDAMENTAL AND MOLECULAR MECHANISMS OF MUTAGENESIS

Continuous single-cell imaging of blood generation from haemogenic endothelium

(2009) Hanna M. Eilken et al.   NATURE

A p53-mediated DNA damage response limits reprogramming to ensure iPS cell genomic integrity

(2009) Rosa M. Marión et al.   NATURE

Suppression of induced pluripotent stem cell generation by the p53–p21 pathway

(2009) Hyenjong Hong et al.   NATURE

Disease-corrected haematopoietic progenitors from Fanconi anaemia induced pluripotent stem cells

(2009) Ángel Raya et al.   NATURE

Forcing cells to change lineages

(2009) Thomas Graf et al.   NATURE

The Ink4/Arf locus is a barrier for iPS cell reprogramming

(2009) Han Li et al.   NATURE

Exploiting and antagonizing microRNA regulation for therapeutic and experimental applications

(2009) Brian D. Brown et al.   NATURE REVIEWS GENETICS

Gene Therapy Fulfilling Its Promise

(2009) Donald B. Kohn et al.   NEW ENGLAND JOURNAL OF MEDICINE

Gene Specificity of Suppression of Transgene-Mediated Insertional Transcriptional Activation by the Chicken HS4 Insulator

(2009) Romain Desprat et al.   PLoS One

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

(2009) N. Cartier et al.   SCIENCE

Bioluminescent Imaging Demonstrates That Transplanted Human Embryonic Stem Cell-Derived CD34+Cells Preferentially Develop into Endothelial Cells

(2009) Xinghui Tian et al.   STEM CELLS

Granulocyte Colony-Stimulating Factor-Based Stem Cell Mobilization in Patients with Sickle Cell Disease

(2008) Cara Rosenbaum et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

Hematopoiesis: An Evolving Paradigm for Stem Cell Biology

(2008) Stuart H. Orkin et al.   CELL

The Emergence of Hematopoietic Stem Cells Is Initiated in the Placental Vasculature in the Absence of Circulation

(2008) Katrin E. Rhodes et al.   Cell Stem Cell

Fate Tracing Reveals the Endothelial Origin of Hematopoietic Stem Cells

(2008) Ann C. Zovein et al.   Cell Stem Cell

Efficient Hematopoietic Differentiation of Human Embryonic Stem Cells on Stromal Cells Derived from Hematopoietic Niches

(2008) Maria H. Ledran et al.   Cell Stem Cell

Mechanisms Governing Lentivirus Integration Site Selection

(2008) Angela Ciuffi   CURRENT GENE THERAPY

Stem Cell Engineering for the Treatment of Severe Hemoglobinopathies

(2008) Michel Sadelain et al.   CURRENT MOLECULAR MEDICINE

Impaired embryonic haematopoiesis yet normal arterial development in the absence of the Notch ligand Jagged1

(2008) Àlex Robert-Moreno et al.   EMBO JOURNAL

High incidence of leukemia in large animals after stem cell gene therapy with a HOXB4-expressing retroviral vector

(2008) Xiao-Bing Zhang et al.   JOURNAL OF CLINICAL INVESTIGATION

Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients

(2008) Steven J. Howe et al.   JOURNAL OF CLINICAL INVESTIGATION

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

(2008) Salima Hacein-Bey-Abina et al.   JOURNAL OF CLINICAL INVESTIGATION

Reduced Genotoxicity of Avian Sarcoma Leukosis Virus Vectors in Rhesus Long-term Repopulating Cells Compared to Standard Murine Retrovirus Vectors

(2008) Jingqiong Hu et al.   MOLECULAR THERAPY

Highly efficient transient gene expression and gene targeting in primate embryonic stem cells with helper-dependent adenoviral vectors

(2008) K. Suzuki et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA