An ex vivo gene therapy approach to treat muscular dystrophy using inducible pluripotent stem cells

Genetic correction of β-thalassemia patient-specific iPS cells and its use in improving hemoglobin production in irradiated SCID mice

(2012) Yixuan Wang et al.   CELL RESEARCH

Human ES- and iPS-Derived Myogenic Progenitors Restore DYSTROPHIN and Improve Contractility upon Transplantation in Dystrophic Mice

(2012) Radbod Darabi et al.   Cell Stem Cell

Transplantation of Genetically Corrected Human iPSC-Derived Progenitors in Mice with Limb-Girdle Muscular Dystrophy

(2012) F. S. Tedesco et al.   Science Translational Medicine

Stem Cell-Mediated Transfer of a Human Artificial Chromosome Ameliorates Muscular Dystrophy

(2011) F. S. Tedesco et al.   Science Translational Medicine

Functional Myogenic Engraftment from Mouse iPS Cells

(2011) Radbod Darabi et al.   Stem Cell Reviews and Reports

Assessment of the Myogenic Stem Cell Compartment Following Transplantation ofPax3/Pax7-Induced Embryonic Stem Cell-Derived Progenitors

(2011) Radbod Darabi et al.   STEM CELLS

Intrinsic cell memory reinforces myogenic commitment of pericyte-derived iPSCs

(2010) Mattia Quattrocelli et al.   JOURNAL OF PATHOLOGY

Aberrant silencing of imprinted genes on chromosome 12qF1 in mouse induced pluripotent stem cells

(2010) Matthias Stadtfeld et al.   NATURE

Molecular evolution of a novel hyperactive Sleeping Beauty transposase enables robust stable gene transfer in vertebrates

(2009) Lajos Mátés et al.   NATURE GENETICS

Functional Substitution by TAT-Utrophin in Dystrophin-Deficient Mice

(2009) Kevin J. Sonnemann et al.   PLOS MEDICINE

c-Myc Is Dispensable for Direct Reprogramming of Mouse Fibroblasts

(2008) Marius Wernig et al.   Cell Stem Cell

Microutrophin Delivery Through rAAV6 Increases Lifespan and Improves Muscle Function in Dystrophic Dystrophin/Utrophin-deficient Mice

(2008) Guy L Odom et al.   MOLECULAR THERAPY

Functional skeletal muscle regeneration from differentiating embryonic stem cells

(2008) Radbod Darabi et al.   NATURE MEDICINE