Genomic integration of the full-length dystrophin coding sequence in Duchenne muscular dystrophy induced pluripotent stem cells

HEK293 cells express dystrophin Dp71 with nucleus-specific localization of Dp71ab

(2016) Atsushi Nishida et al.   HISTOCHEMISTRY AND CELL BIOLOGY

Gene therapies that restore dystrophin expression for the treatment of Duchenne muscular dystrophy

(2016) Jacqueline N. Robinson-Hamm et al.   HUMAN GENETICS

Precise Correction of Disease Mutations in Induced Pluripotent Stem Cells Derived From Patients With Limb Girdle Muscular Dystrophy

(2016) Soeren Turan et al.   MOLECULAR THERAPY

The Pathogenesis and Therapy of Muscular Dystrophies

(2015) Simon Guiraud et al.   Annual Review of Genomics and Human Genetics

Progress and prospects of gene therapy clinical trials for the muscular dystrophies

(2015) Niclas E. Bengtsson et al.   HUMAN MOLECULAR GENETICS

piggyBactransposons expressing full-length human dystrophin enable genetic correction of dystrophic mesoangioblasts

(2015) Mariana Loperfido et al.   NUCLEIC ACIDS RESEARCH

Recombinase-Mediated Reprogramming and Dystrophin Gene Addition in mdx Mouse Induced Pluripotent Stem Cells

(2014) Chunli Zhao et al.   PLoS One

Triple Trans-Splicing Adeno-Associated Virus Vectors Capable of Transferring the Coding Sequence for Full-Length Dystrophin Protein into Dystrophic Mice

(2013) Taeyoung Koo et al.   HUMAN GENE THERAPY

Muscular dystrophies

(2013) Eugenio Mercuri et al.   LANCET

DICE, an efficient system for iterative genomic editing in human pluripotent stem cells

(2013) Fangfang Zhu et al.   NUCLEIC ACIDS RESEARCH

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

(2013) Antonio Filareto et al.   Nature Communications

Therapeutic Applications of the PhiC31 Integrase System

(2012) Christopher L. Chavez et al.   CURRENT GENE THERAPY

Functional Efficacy of Dystrophin Expression from Plasmids Delivered to mdx Mice by Hydrodynamic Limb Vein Injection

(2009) Guofeng Zhang et al.   HUMAN GENE THERAPY

Improving human interferon-β production in mammalian cell lines by insertion of an intronic sequence within its naturally uninterrupted gene

(2008) Paola Zago et al.   BIOTECHNOLOGY AND APPLIED BIOCHEMISTRY