iPSCs as a Platform for Disease Modeling, Drug Screening, and Personalized Therapy in Muscular Dystrophies

Tracking progress: an update on animal models for Duchenne muscular dystrophy

(2018) Dominic J. Wells   Disease Models & Mechanisms

Recapitulating muscle disease phenotypes with myotonic dystrophy 1 induced pluripotent stem cells: a tool for disease modeling and drug discovery

(2018) Ricardo Mondragon-Gonzalez et al.   Disease Models & Mechanisms

Three-Dimensional Human iPSC-Derived Artificial Skeletal Muscles Model Muscular Dystrophies and Enable Multilineage Tissue Engineering

(2018) Sara Martina Maffioletti et al.   Cell Reports

Correction of diverse muscular dystrophy mutations in human engineered heart muscle by single-site genome editing

(2018) Chengzu Long et al.   Science Advances

Nanotopography-responsive myotube alignment and orientation as a sensitive phenotypic biomarker for Duchenne Muscular Dystrophy

(2018) Bin Xu et al.   BIOMATERIALS

A Myogenic Double-Reporter Human Pluripotent Stem Cell Line Allows Prospective Isolation of Skeletal Muscle Progenitors

(2018) Jianbo Wu et al.   Cell Reports

Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

(2017) Craig M McDonald et al.   LANCET

ERBB3 and NGFR mark a distinct skeletal muscle progenitor cell in human development and hPSCs

(2017) Michael R. Hicks et al.   NATURE CELL BIOLOGY

A human iPS cell myogenic differentiation system permitting high-throughput drug screening

(2017) Tomoya Uchimura et al.   Stem Cell Research

PAX7 Targets, CD54, Integrin α9β1, and SDC2, Allow Isolation of Human ESC/iPSC-Derived Myogenic Progenitors

(2017) Alessandro Magli et al.   Cell Reports

Expansion and Purification Are Critical for the Therapeutic Application of Pluripotent Stem Cell-Derived Myogenic Progenitors

(2017) Jaemin Kim et al.   Stem Cell Reports

CRISPR-Cpf1 correction of muscular dystrophy mutations in human cardiomyocytes and mice

(2017) Yu Zhang et al.   Science Advances

A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells

(2016) Courtney S. Young et al.   Cell Stem Cell

Nicorandil, a Nitric Oxide Donor and ATP-Sensitive Potassium Channel Opener, Protects Against Dystrophin-Deficient Cardiomyopathy

(2016) Muhammad Z. Afzal et al.   JOURNAL OF CARDIOVASCULAR PHARMACOLOGY AND THERAPEUTICS

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

Concordant but Varied Phenotypes among Duchenne Muscular Dystrophy Patient-Specific Myoblasts Derived using a Human iPSC-Based Model

(2016) In Young Choi et al.   Cell Reports

Modeling and study of the mechanism of dilated cardiomyopathy using induced pluripotent stem cells derived from individuals with Duchenne muscular dystrophy

(2015) B. Lin et al.   Disease Models & Mechanisms

Skeletal muscle perfusion and stem cell delivery in muscle disorders using intra-femoral artery canulation in mice

(2015) Nadine Matthias et al.   EXPERIMENTAL CELL RESEARCH

Porcine Models of Muscular Dystrophy

(2015) J. T. Selsby et al.   ILAR JOURNAL

Differentiation of pluripotent stem cells to muscle fiber to model Duchenne muscular dystrophy

(2015) Jérome Chal et al.   NATURE BIOTECHNOLOGY

Early pathogenesis of Duchenne muscular dystrophy modelled in patient-derived human induced pluripotent stem cells

(2015) Emi Shoji et al.   Scientific Reports

Precise Correction of the Dystrophin Gene in Duchenne Muscular Dystrophy Patient Induced Pluripotent Stem Cells by TALEN and CRISPR-Cas9

(2015) Hongmei Lisa Li et al.   Stem Cell Reports

Improving CRISPR-Cas nuclease specificity using truncated guide RNAs

(2014) Yanfang Fu et al.   NATURE BIOTECHNOLOGY

Duchenne and Becker Muscular Dystrophies

(2014) Kevin M. Flanigan   NEUROLOGIC CLINICS

Myogenic Differentiation of Muscular Dystrophy-Specific Induced Pluripotent Stem Cells for Use in Drug Discovery

(2014) Ramzey Abujarour et al.   Stem Cells Translational Medicine

Derivation and Expansion of PAX7-Positive Muscle Progenitors from Human and Mouse Embryonic Stem Cells

(2014) Michael Shelton et al.   Stem Cell Reports

Double Nicking by RNA-Guided CRISPR Cas9 for Enhanced Genome Editing Specificity

(2013) F. Ann Ran et al.   CELL

Immunogenicity of Pluripotent Stem Cells and Their Derivatives

(2013) Patricia E. de Almeida et al.   CIRCULATION RESEARCH

Comparative Genomics of X-linked Muscular Dystrophies: The Golden Retriever Model

(2013) Candice Brinkmeyer-Langford et al.   CURRENT GENOMICS

Length-dependent CTG{middle dot}CAG triplet-repeat expansion in myotonic dystrophy patient-derived induced pluripotent stem cells

(2013) J. Du et al.   HUMAN MOLECULAR GENETICS

Muscular dystrophies

(2013) Eugenio Mercuri et al.   LANCET

High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells

(2013) Yanfang Fu et al.   NATURE BIOTECHNOLOGY

Muscular Dystrophies and Other Genetic Myopathies

(2013) Perry B. Shieh   NEUROLOGIC CLINICS

Correction: Efficient and Reproducible Myogenic Differentiation from Human iPS Cells: Prospects for Modeling Miyoshi Myopathy In Vitro

(2013) Akihito Tanaka et al.   PLoS One

RNA-Guided Human Genome Engineering via Cas9

(2013) P. Mali et al.   SCIENCE

Multiplex Genome Engineering Using CRISPR/Cas Systems

(2013) L. Cong et al.   SCIENCE

Exon skipping and gene transfer restore dystrophin expression in hiPSC-cardiomyocytes harbouring DMD mutations

(2013) Emily Dick et al.   STEM CELLS AND DEVELOPMENT

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

(2013) Antonio Filareto et al.   Nature Communications

Derivation and FACS-Mediated Purification of PAX3+/PAX7+ Skeletal Muscle Precursors from Human Pluripotent Stem Cells

(2013) Bianca Borchin et al.   Stem Cell Reports

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

Structural Basis for Sequence-Specific Recognition of DNA by TAL Effectors

(2012) D. Deng et al.   SCIENCE

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

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

Revealing off-target cleavage specificities of zinc-finger nucleases by in vitro selection

(2011) Vikram Pattanayak et al.   NATURE METHODS

Functional Myogenic Engraftment from Mouse iPS Cells

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

Genetic Background Affects Properties of Satellite Cells and mdx Phenotypes

(2010) So-ichiro Fukada et al.   AMERICAN JOURNAL OF PATHOLOGY

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

(2010) Luigi Warren et al.   Cell Stem Cell

Recent Advances in Lentiviral Vector Development and Applications

(2010) Janka Mátrai et al.   MOLECULAR THERAPY

Facioscapulohumeral Dystrophy: Incomplete Suppression of a Retrotransposed Gene

(2010) Lauren Snider et al.   PLoS Genetics

The effects of cardioactive drugs on cardiomyocytes derived from human induced pluripotent stem cells

(2009) Noritaka Yokoo et al.   BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS

Parkinson's Disease Patient-Derived Induced Pluripotent Stem Cells Free of Viral Reprogramming Factors

(2009) Frank Soldner et al.   CELL

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

(2009) Dohoon Kim et al.   Cell Stem Cell

Complete Genetic Correction of iPS Cells From Duchenne Muscular Dystrophy

(2009) Yasuhiro Kazuki et al.   MOLECULAR THERAPY

Efficient induction of transgene-free human pluripotent stem cells using a vector based on Sendai virus, an RNA virus that does not integrate into the host genome

(2009) Noemi FUSAKI et al.   PROCEEDINGS OF THE JAPAN ACADEMY SERIES B-PHYSICAL AND BIOLOGICAL SCIENCES

Generation of mouse-induced pluripotent stem cells by transient expression of a single nonviral polycistronic vector

(2009) F. Gonzalez et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

A Simple Cipher Governs DNA Recognition by TAL Effectors

(2009) M. J. Moscou et al.   SCIENCE

Disease-Specific Induced Pluripotent Stem Cells

(2008) In-Hyun Park et al.   CELL

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

(2008) Salima Hacein-Bey-Abina et al.   JOURNAL OF CLINICAL INVESTIGATION

Time to reconsider stem cell ethics--the importance of induced pluripotent cells

(2008) S. Holm   JOURNAL OF MEDICAL ETHICS

Generation of Mouse Induced Pluripotent Stem Cells Without Viral Vectors

(2008) K. Okita et al.   SCIENCE

Pulmonary Passage is a Major Obstacle for Intravenous Stem Cell Delivery: The Pulmonary First-Pass Effect

(2008) Uwe M. Fischer et al.   STEM CELLS AND DEVELOPMENT

Alternative sources of pluripotency: science, ethics, and stem cells

(2008) Zachary J. Kastenberg et al.   Transplantation Reviews