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

Efficient human iPS cell derivation by a non-integrating plasmid from blood cells with unique epigenetic and gene expression signatures

(2011) Bin-Kuan Chou et al.   CELL RESEARCH

A self-inactivating lentiviral vector for SCID-X1 gene therapy that does not activate LMO2 expression in human T cells

(2010) S. Zhou et al.   BLOOD

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

(2010) Luigi Warren et al.   Cell Stem Cell

Functional genomics, proteomics, and regulatory DNA analysis in isogenic settings using zinc finger nuclease-driven transgenesis into a safe harbor locus in the human genome

(2010) R. C. DeKelver et al.   GENOME RESEARCH

Hematopoietic Stem Cell Transplantation for Chronic Granulomatous Disease

(2010) Reinhard A. Seger   IMMUNOLOGY AND ALLERGY CLINICS OF NORTH AMERICA

Patient-specific induced pluripotent stem-cell-derived models of LEOPARD syndrome

(2010) Xonia Carvajal-Vergara 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

Human-induced pluripotent stem cells from blood cells of healthy donors and patients with acquired blood disorders

(2009) Z. Ye 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

Derivation of functional mature neutrophils from human embryonic stem cells

(2009) Y. Yokoyama et al.   BLOOD

Toward clinical therapies using hematopoietic cells derived from human pluripotent stem cells

(2009) D. S. Kaufman   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

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

Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases

(2009) Dirk Hockemeyer et al.   NATURE BIOTECHNOLOGY

Chronic Granulomatous Disease: The European Experience

(2009) J. Merlijn van den Berg et al.   PLoS One

Site-specific integration of adeno-associated virus involves partial duplication of the target locus

(2009) E. Henckaerts et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

Induced pluripotent stem cells from a spinal muscular atrophy patient

(2008) Allison D. Ebert et al.   NATURE

Robust, Persistent Transgene Expression in Human Embryonic Stem Cells Is Achieved with AAVS1-Targeted Integration

(2007) Joseph R. Smith et al.   STEM CELLS