Stem cell gene therapy: the risks of insertional mutagenesis and approaches to minimize genotoxicity

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

Oxidase-deficient neutrophils from X-linked chronic granulomatous disease iPS cells: functional correction by zinc finger nuclease-mediated safe harbor targeting

(2011) J. Zou et al.   BLOOD

Human Induced Pluripotent Stem Cells: The Past, Present, and Future

(2011) S M I Hussein et al.   CLINICAL PHARMACOLOGY & THERAPEUTICS

Genotoxicity of retroviral hematopoietic stem cell gene therapy

(2011) Grant D Trobridge   EXPERT OPINION ON BIOLOGICAL THERAPY

Safeguarding Nonhuman Primate iPS Cells With Suicide Genes

(2011) Bonan Zhong et al.   MOLECULAR THERAPY

Hotspots of aberrant epigenomic reprogramming in human induced pluripotent stem cells

(2011) Ryan Lister et al.   NATURE

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

(2011) Troy Brady et al.   NUCLEIC ACIDS RESEARCH

Human CD34+ CD133+ Hematopoietic Stem Cells Cultured with Growth Factors Including Angptl5 Efficiently Engraft Adult NOD-SCID Il2rγ−/− (NSG) Mice

(2011) Adam C. Drake et al.   PLoS One

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

(2010) Luigi Warren et al.   Cell Stem Cell

Integration profile of retroviral vector in gene therapy treated patients is cell-specific according to gene expression and chromatin conformation of target cell

(2010) Luca Biasco et al.   EMBO Molecular Medicine

Gene therapy for β-thalassaemia: the continuing challenge

(2010) Evangelia Yannaki et al.   EXPERT REVIEWS IN MOLECULAR MEDICINE

Large animal models of hematopoietic stem cell gene therapy

(2010) G D Trobridge et al.   GENE THERAPY

Clinical Impact of Suicide Gene Therapy in Allogeneic Hematopoietic Stem Cell Transplantation

(2010) Maria Teresa Lupo-Stanghellini et al.   HUMAN GENE THERAPY

Inhibition of In Vivo HIV Infection in Humanized Mice by Gene Therapy of Human Hematopoietic Stem Cells with a Lentiviral Vector Encoding a Broadly Neutralizing Anti-HIV Antibody

(2010) A. Joseph et al.   JOURNAL OF VIROLOGY

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

(2010) Nicole M Kane et al.   MOLECULAR THERAPY

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

Lentiviral Vector Gene Therapy: Effective and Safe?

(2010) Derek A Persons   MOLECULAR THERAPY

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

(2010) Marina Cavazzana-Calvo et al.   NATURE

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

(2010) Eirini P Papapetrou et al.   NATURE BIOTECHNOLOGY

20 years of gene therapy for SCID

(2010) Alain Fischer et al.   NATURE IMMUNOLOGY

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

Efficacy of Gene Therapy for X-Linked Severe Combined Immunodeficiency

(2010) Salima Hacein-Bey-Abina et al.   NEW ENGLAND JOURNAL OF MEDICINE

Stem-Cell Gene Therapy for the Wiskott–Aldrich Syndrome

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

No Evidence for Clonal Selection Due to Lentiviral Integration Sites in Human Induced Pluripotent Stem Cells

(2010) Thomas Winkler et al.   STEM CELLS

HIV integration site distributions in resting and activated CD4+ T cells infected in culture

(2009) Troy Brady et al.   AIDS

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

Integration of retroviral vectors induces minor changes in the transcriptional activity of T cells from ADA-SCID patients treated with gene therapy

(2009) B. Cassani et al.   BLOOD

Retrovirus gene therapy for X-linked chronic granulomatous disease can achieve stable long-term correction of oxidase activity in peripheral blood neutrophils

(2009) E. M. Kang et al.   BLOOD

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

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

(2009) Ute Modlich et al.   MOLECULAR THERAPY

Virus-free induction of pluripotency and subsequent excision of reprogramming factors

(2009) Keisuke Kaji 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

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

(2009) N. Cartier et al.   SCIENCE

Resistance of mature T cells to oncogene transformation

(2008) S. Newrzela et al.   BLOOD

Flanking-sequence exponential anchored–polymerase chain reaction amplification: a sensitive and highly specific method for detecting retroviral integrant–host–junction sequences

(2008) M.A. Pule et al.   CYTOTHERAPY

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

Physiological Promoters Reduce the Genotoxic Risk of Integrating Gene Vectors

(2008) Daniela Zychlinski et al.   MOLECULAR THERAPY

DNA bar coding and pyrosequencing to analyze adverse events in therapeutic gene transfer

(2008) Gary P. Wang et al.   NUCLEIC ACIDS RESEARCH

Induced Pluripotent Stem Cells Generated Without Viral Integration

(2008) M. Stadtfeld et al.   SCIENCE

Generation of Mouse Induced Pluripotent Stem Cells Without Viral Vectors

(2008) K. Okita et al.   SCIENCE

Induced Pluripotent Stem Cell Generation Using a Single Lentiviral Stem Cell Cassette

(2008) Cesar A. Sommer et al.   STEM CELLS