Gene therapy for β-thalassaemia: the continuing challenge

Hematopoietic stem cell mobilization strategies for gene therapy of beta thalassemia and sickle cell disease

(2010) Evangelia Yannaki et al.   Annals of the New York Academy of Sciences

Current status of globin gene therapy for the treatment of β-thalassaemia

(2010) Leszek Lisowski et al.   BRITISH JOURNAL OF HAEMATOLOGY

Induction of pluripotency by defined factors

(2010) Keisuke Okita et al.   EXPERIMENTAL CELL RESEARCH

Induced pluripotent stem cells – alchemist's tale or clinical reality?

(2010) S. Tamir Rashid et al.   EXPERT REVIEWS IN MOLECULAR MEDICINE

Progress toward the clinical application of patient-specific pluripotent stem cells

(2010) Evangelos Kiskinis et al.   JOURNAL OF CLINICAL INVESTIGATION

Nuclear reprogramming to a pluripotent state by three approaches

(2010) Shinya Yamanaka et al.   NATURE

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

Advances in Reprogramming Somatic Cells to Induced Pluripotent Stem Cells

(2010) Minal Patel et al.   Stem Cell Reviews and Reports

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

A novel human gamma-globin gene vector for genetic correction of sickle cell anemia in a humanized sickle mouse model: critical determinants for successful correction

(2009) A. Perumbeti et al.   BLOOD

Amelioration of murine  -thalassemia through drug selection of hematopoietic stem cells transduced with a lentiviral vector encoding both  -globin and the MGMT drug-resistance gene

(2009) H. Zhao et al.   BLOOD

How I treat ADA deficiency

(2009) H. B. Gaspar et al.   BLOOD

Generation of Human Induced Pluripotent Stem Cells by Direct Delivery of Reprogramming Proteins

(2009) Dohoon Kim et al.   Cell Stem Cell

Mobilization of Hematopoietic Stem Cells in a Thalassemic Mouse Model: Implications for Human Gene Therapy of Thalassemia

(2009) Evangelia Yannaki et al.   HUMAN 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

Genotoxic Potential of Lineage-specific Lentivirus Vectors Carrying the β-Globin Locus Control Region

(2009) Paritha I Arumugam 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

Modelling pathogenesis and treatment of familial dysautonomia using patient-specific iPSCs

(2009) Gabsang Lee et al.   NATURE

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

(2009) Ángel Raya et al.   NATURE

Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency

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

Induced pluripotent stem cells offer new approach to therapy in thalassemia and sickle cell anemia and option in prenatal diagnosis in genetic diseases

(2009) L. Ye et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

 -Thalassemia Treatment Succeeds, With a Caveat

(2009) J. Kaiser   SCIENCE

Human Induced Pluripotent Stem Cells Free of Vector and Transgene Sequences

(2009) J. Yu et al.   SCIENCE

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

(2009) N. Cartier et al.   SCIENCE

Hematopoietic stem cell gene transfer for the treatment of hemoglobin disorders

(2009) D. A. Persons   Hematology-American Society of Hematology Education Program

Ten years of gene therapy for primary immune deficiencies

(2009) A. Aiuti et al.   Hematology-American Society of Hematology Education Program

Disease-Specific Induced Pluripotent Stem Cells

(2008) In-Hyun Park et al.   CELL

Integrase, LEDGF/p75 and HIV replication

(2008) E. M. Poeschla   CELLULAR AND MOLECULAR LIFE SCIENCES

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

Physiological Promoters Reduce the Genotoxic Risk of Integrating Gene Vectors

(2008) Daniela Zychlinski et al.   MOLECULAR THERAPY

Globin Lentiviral Vector Insertions Can Perturb the Expression of Endogenous Genes in β-thalassemic Hematopoietic Cells

(2008) Phillip W Hargrove et al.   MOLECULAR THERAPY

Induced pluripotent stem cells from a spinal muscular atrophy patient

(2008) Allison D. Ebert et al.   NATURE

In vivo selection of genetically modified erythroblastic progenitors leads to long-term correction of  -thalassemia

(2008) A. Miccio et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Combinatorial Incorporation of Enhancer-Blocking Components of the Chicken β-Globin 5′HS4 and Human T-Cell Receptor α/δ BEAD-1 Insulators in Self-Inactivating Retroviral Vectors Reduces Their Genotoxic Potential

(2008) Ali Ramezani et al.   STEM CELLS

Advances in the allogeneic transplantation for thalassemia

(2007) Guido Lucarelli et al.   BLOOD REVIEWS