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

Globin phenotype of erythroid cells derived from human induced pluripotent stem cells

(2010) K.-H. Chang et al.   BLOOD

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

(2010) Marina Cavazzana-Calvo et al.   NATURE

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

Characterization of human embryonic stem cells with features of neoplastic progression

(2009) Tamra E Werbowetski-Ogilvie et al.   NATURE BIOTECHNOLOGY

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

(2009) Dirk Hockemeyer et al.   NATURE BIOTECHNOLOGY

Stoichiometric and temporal requirements of Oct4, Sox2, Klf4, and c-Myc expression for efficient human iPSC induction and differentiation

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

Genetic Modification of Human Embryonic Stem Cells for Derivation of Target Cells

(2008) Antonietta Giudice et al.   Cell Stem Cell

Stem Cell Engineering for the Treatment of Severe Hemoglobinopathies

(2008) Michel Sadelain et al.   CURRENT MOLECULAR MEDICINE

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

OP9 Stroma Augments Survival of Hematopoietic Precursors and Progenitors During Hematopoietic Differentiation from Human Embryonic Stem Cells

(2008) Junfeng Ji et al.   STEM CELLS

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

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