Concise Review: Managing Genotoxicity in the Therapeutic Modification of Stem Cells

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

Polyclonal fluctuation of lentiviral vector-transduced and expanded murine hematopoietic stem cells

(2011) T. Maetzig et al.   BLOOD

Is normal hematopoiesis maintained solely by long-term multipotent stem cells?

(2011) M. Cavazzana-Calvo et al.   BLOOD

Prdm16 is a physiologic regulator of hematopoietic stem cells

(2011) F. Aguilo 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

Highly Efficient miRNA-Mediated Reprogramming of Mouse and Human Somatic Cells to Pluripotency

(2011) Frederick Anokye-Danso et al.   Cell Stem Cell

Zinc-Finger Nuclease Based Genome Surgery: Its All About Specificity

(2011) Eva-Maria Handel et al.   CURRENT GENE THERAPY

Exploring refined conditions for reprogramming cells by recombinant Oct4 protein

(2011) Marc Thier et al.   INTERNATIONAL JOURNAL OF DEVELOPMENTAL BIOLOGY

Lentiviral Vector Design and Imaging Approaches to Visualize the Early Stages of Cellular Reprogramming

(2011) Eva Warlich et al.   MOLECULAR THERAPY

Copy number variation and selection during reprogramming to pluripotency

(2011) Samer M. Hussein et al.   NATURE

The dark side of induced pluripotency

(2011) Martin F. Pera   NATURE

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

(2011) Troy Brady et al.   NUCLEIC ACIDS RESEARCH

High-definition mapping of retroviral integration sites identifies active regulatory elements in human multipotent hematopoietic progenitors

(2010) C. Cattoglio et al.   BLOOD

Dynamics of gene-modified progenitor cells analyzed by tracking retroviral integration sites in a human SCID-X1 gene therapy trial

(2010) G. P. Wang et al.   BLOOD

Pluripotency and Cellular Reprogramming: Facts, Hypotheses, Unresolved Issues

(2010) Jacob H. Hanna et al.   CELL

Expansion of Human Cord Blood Hematopoietic Stem Cells for Transplantation

(2010) Song Chou 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

The Molecular Basis of Lmo2-Induced T-Cell Acute Lymphoblastic Leukemia

(2010) D. J. Curtis et al.   CLINICAL CANCER RESEARCH

Large animal models of hematopoietic stem cell gene therapy

(2010) G D Trobridge et al.   GENE THERAPY

Effect of the Internal Promoter on Insertional Gene Activation by Lentiviral Vectors with an Intact HIV Long Terminal Repeat

(2010) S. Knight et al.   JOURNAL OF VIROLOGY

AML xenograft efficiency is significantly improved in NOD/SCID-IL2RG mice constitutively expressing human SCF, GM-CSF and IL-3

(2010) M Wunderlich et al.   LEUKEMIA

Ex Vivo Expansion of Retrovirally Transduced Primate CD34+ Cells Results in Overrepresentation of Clones With MDS1/EVI1 Insertion Sites in the Myeloid Lineage After Transplantation

(2010) Stephanie Sellers et al.   MOLECULAR THERAPY

Genetic variegation of clonal architecture and propagating cells in leukaemia

(2010) Kristina Anderson et al.   NATURE

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

(2010) Marina Cavazzana-Calvo et al.   NATURE

High-resolution DNA analysis of human embryonic stem cell lines reveals culture-induced copy number changes and loss of heterozygosity

(2010) Elisa Närvä et al.   NATURE BIOTECHNOLOGY

Gene therapy activates EVI1, destabilizes chromosomes

(2010) Cynthia E Dunbar et al.   NATURE MEDICINE

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

Promotion of direct reprogramming by transformation-deficient Myc

(2010) M. Nakagawa et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Protein transduction from retroviral Gag precursors

(2010) Christine Voelkel et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

(2010) Anthony E. Boitano et al.   SCIENCE

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

Self-inactivating Retroviral Vector-mediated Gene Transfer Induces Oncogene Activation and Immortalization of Primary Murine Bone Marrow Cells

(2009) Marita Bosticardo et al.   MOLECULAR THERAPY

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

(2009) Ute Modlich et al.   MOLECULAR THERAPY

Cell-intrinsic and Vector-related Properties Cooperate to Determine the Incidence and Consequences of Insertional Mutagenesis

(2009) Olga S Kustikova 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

piggyBac transposition reprograms fibroblasts to induced pluripotent stem cells

(2009) Knut Woltjen 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

Comprehensive genomic access to vector integration in clinical gene therapy

(2009) Richard Gabriel et al.   NATURE MEDICINE

Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency

(2009) Alessandro Aiuti et al.   NEW ENGLAND JOURNAL OF MEDICINE

Polycistronic Lentiviral Vector for “Hit and Run” Reprogramming of Adult Skin Fibroblasts to Induced Pluripotent Stem Cells

(2009) Chia-Wei Chang et al.   STEM CELLS

Resistance of mature T cells to oncogene transformation

(2008) S. Newrzela et al.   BLOOD

Evi-1 Is a Critical Regulator for Hematopoietic Stem Cells and Transformed Leukemic Cells

(2008) Susumu Goyama et al.   Cell Stem Cell

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

Leukemia induction after a single retroviral vector insertion in Evi1 or Prdm16

(2008) U Modlich et al.   LEUKEMIA

Human ESCs predisposition to karyotypic instability: Is a matter of culture adaptation or differential vulnerability among hESC lines due to inherent properties?

(2008) Puri Catalina et al.   Molecular Cancer

In Vivo Biosafety Model to Assess the Risk of Adverse Events From Retroviral and Lentiviral Vectors

(2008) Gerhard Bauer et al.   MOLECULAR THERAPY

Generation of Mouse Induced Pluripotent Stem Cells Without Viral Vectors

(2008) K. Okita et al.   SCIENCE