Human iPSC Models to Study Orphan Diseases: Muscular Dystrophies

[1]  T. Cooper,et al.  Mechanisms of skeletal muscle wasting in a mouse model for myotonic dystrophy type 1 , 2018, Human molecular genetics.

[2]  K. Gawlik.  At the Crossroads of Clinical and Preclinical Research for Muscular Dystrophy—Are We Closer to Effective Treatment for Patients? , 2018, International journal of molecular sciences.

[3]  J. Utikal,et al.  Ion Channel Dysfunctions in Dilated Cardiomyopathy in Limb-Girdle Muscular Dystrophy , 2018, Circulation. Genomic and precision medicine.

[4]  Malte Tiburcy,et al.  Correction of diverse muscular dystrophy mutations in human engineered heart muscle by single-site genome editing , 2018, Science Advances.

[5]  H. Sakurai,et al.  A human iPS cell myogenic differentiation system permitting high-throughput drug screening. , 2017, Stem cell research.

[6]  G. Meola,et al.  CRISPR/Cas9-Mediated Deletion of CTG Expansions Recovers Normal Phenotype in Myogenic Cells Derived from Myotonic Dystrophy 1 Patients , 2017, Molecular therapy. Nucleic acids.

[7]  C. Paradas,et al.  Generation of an induced pluripotent stem cell line (CSCRMi001-A) from a patient with a new type of limb-girdle muscular dystrophy (LGMD) due to a missense mutation in POGLUT1 (Rumi). , 2017, Stem Cell Research.

[8]  S. Takeda,et al.  Skeletal muscle generated from induced pluripotent stem cells - induction and application , 2017, World journal of stem cells.

[9]  P. Gee,et al.  Cellular Reprogramming, Genome Editing, and Alternative CRISPR Cas9 Technologies for Precise Gene Therapy of Duchenne Muscular Dystrophy , 2017, Stem cells international.

[10]  Dharmendra Kumar,et al.  Clinical potential of human-induced pluripotent stem cells , 2017, Cell Biology and Toxicology.

[11]  Takashi Daimon,et al.  Autologous Induced Stem‐Cell–Derived Retinal Cells for Macular Degeneration: Brief Report , 2017, The New England journal of medicine.

[12]  Š. Polák,et al.  iPS cell technologies and their prospect for bone regeneration and disease modeling: A mini review , 2017, Journal of advanced research.

[13]  M. Fehlings,et al.  Induced Pluripotent Stem Cells for Traumatic Spinal Cord Injury , 2017, Front. Cell Dev. Biol..

[14]  M. Willemse,et al.  CRISPR/Cas9-Induced (CTG⋅CAG)n Repeat Instability in the Myotonic Dystrophy Type 1 Locus: Implications for Therapeutic Genome Editing , 2017, Molecular therapy : the journal of the American Society of Gene Therapy.

[15]  E. Pareja,et al.  Clinical Application of Pluripotent Stem Cells: An Alternative Cell-Based Therapy for Treating Liver Diseases? , 2016, Transplantation.

[16]  L. D. Del Priore,et al.  Potential of Induced Pluripotent Stem Cells (iPSCs) for Treating Age-Related Macular Degeneration (AMD) , 2016, Cells.

[17]  G. Pita,et al.  A POGLUT1 mutation causes a muscular dystrophy with reduced Notch signaling and satellite cell loss , 2016, EMBO molecular medicine.

[18]  P. Sunil.  Induced pluripotent stem cells in dentistry , 2016, Journal of pharmacy & bioallied sciences.

[19]  Olivier Pourquié,et al.  Generation of human muscle fibers and satellite-like cells from human pluripotent stem cells in vitro , 2016, Nature Protocols.

[20]  G. Coppola,et al.  A Novel Protocol for Directed Differentiation of C9orf72-Associated Human Induced Pluripotent Stem Cells Into Contractile Skeletal Myotubes , 2016, Stem cells translational medicine.

[21]  Daniel G. Miller,et al.  A Human Pluripotent Stem Cell Model of Facioscapulohumeral Muscular Dystrophy‐Affected Skeletal Muscles , 2016, Stem Cells Translational Medicine.

[22]  Atsushi Nakano,et al.  A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells. , 2016, Cell stem cell.

[23]  T. Ashizawa,et al.  Genome Therapy of Myotonic Dystrophy Type 1 iPS Cells for Development of Autologous Stem Cell Therapy. , 2016, Molecular therapy : the journal of the American Society of Gene Therapy.

[24]  Soeren Turan,et al.  Precise Correction of Disease Mutations in Induced Pluripotent Stem Cells Derived From Patients With Limb Girdle Muscular Dystrophy. , 2016, Molecular therapy : the journal of the American Society of Gene Therapy.

[25]  J. Vissing,et al.  A New Mouse Model of Limb-Girdle Muscular Dystrophy Type 2I Homozygous for the Common L276I Mutation Mimicking the Mild Phenotype in Humans , 2015, Journal of neuropathology and experimental neurology.

[26]  J. Pomerantz,et al.  Human Satellite Cell Transplantation and Regeneration from Diverse Skeletal Muscles , 2015, Stem cell reports.

[27]  Tom H. Cheung,et al.  Ex Vivo Expansion and In Vivo Self-Renewal of Human Muscle Stem Cells , 2015, Stem cell reports.

[28]  Olivier Tassy,et al.  Differentiation of pluripotent stem cells to muscle fiber to model Duchenne muscular dystrophy , 2015, Nature Biotechnology.

[29]  N. Fujii,et al.  Early pathogenesis of Duchenne muscular dystrophy modelled in patient-derived human induced pluripotent stem cells , 2015, Scientific Reports.

[30]  S. Benedetti,et al.  Corrigendum: Efficient derivation and inducible differentiation of expandable skeletal myogenic cells from human ES and patient-specific iPS cells , 2015, Nature Protocols.

[31]  S. Benedetti,et al.  Efficient derivation and inducible differentiation of expandable skeletal myogenic cells from human ES and patient-specific iPS cells , 2015, Nature Protocols.

[32]  M. Swanson,et al.  Genome Modification Leads to Phenotype Reversal in Human Myotonic Dystrophy Type 1 Induced Pluripotent Stem Cell‐Derived Neural Stem Cells , 2015, Stem cells.

[33]  G. Bett,et al.  Modeling and study of the mechanism of dilated cardiomyopathy using induced pluripotent stem cells derived from individuals with Duchenne muscular dystrophy , 2015, Disease Models & Mechanisms.

[34]  Katherine A James,et al.  Prevalence of Duchenne and Becker Muscular Dystrophies in the United States , 2015, Pediatrics.

[35]  M. Edel,et al.  Myogenic Precursors from iPS Cells for Skeletal Muscle Cell Replacement Therapy , 2015, Journal of clinical medicine.

[36]  Tetsushi Sakuma,et al.  Precise Correction of the Dystrophin Gene in Duchenne Muscular Dystrophy Patient Induced Pluripotent Stem Cells by TALEN and CRISPR-Cas9 , 2014, Stem cell reports.

[37]  D. Hilton‐Jones,et al.  Myotonic dystrophy: diagnosis, management and new therapies. , 2014, Current opinion in neurology.

[38]  W. Stanford,et al.  Derivation and Expansion of PAX7-Positive Muscle Progenitors from Human and Mouse Embryonic Stem Cells , 2014, Stem cell reports.

[39]  M. Rudnicki,et al.  Notch signaling deficiency underlies age-dependent depletion of satellite cells in muscular dystrophy , 2014, Disease Models & Mechanisms.

[40]  Masatoshi Suzuki,et al.  Derivation of Myogenic Progenitors Directly From Human Pluripotent Stem Cells Using a Sphere‐Based Culture , 2014, Stem cells translational medicine.

[41]  Bahram Valamehr,et al.  Myogenic Differentiation of Muscular Dystrophy‐Specific Induced Pluripotent Stem Cells for Use in Drug Discovery , 2014, Stem cells translational medicine.

[42]  Bolan Yu,et al.  Modeling induced pluripotent stem cells from fibroblasts of Duchenne muscular dystrophy patients , 2014, The International journal of neuroscience.

[43]  Joseph Chen,et al.  Derivation and FACS-Mediated Purification of PAX3+/PAX7+ Skeletal Muscle Precursors from Human Pluripotent Stem Cells , 2013, Stem cell reports.

[44]  N. Fujii,et al.  Efficient and Reproducible Myogenic Differentiation from Human iPS Cells: Prospects for Modeling Miyoshi Myopathy In Vitro , 2013, PloS one.

[45]  M. Swanson,et al.  Generation of neural cells from DM1 induced pluripotent stem cells as cellular model for the study of central nervous system neuropathogenesis. , 2013, Cellular reprogramming.

[46]  F. Metzger,et al.  Molecular, Physiological, and Motor Performance Defects in DMSXL Mice Carrying >1,000 CTG Repeats from the Human DM1 Locus , 2012, PLoS genetics.

[47]  K. Wilschut,et al.  Concise Review: Stem Cell Therapy for Muscular Dystrophies , 2012, Stem cells translational medicine.

[48]  R. Krahe,et al.  The myotonic dystrophies: molecular, clinical, and therapeutic challenges , 2012, The Lancet Neurology.

[49]  J. Rousseau,et al.  Myoblasts Derived From Normal hESCs and Dystrophic hiPSCs Efficiently Fuse With Existing Muscle Fibers Following Transplantation , 2012, Molecular therapy : the journal of the American Society of Gene Therapy.

[50]  M. Ares,et al.  Muscleblind-like 2-Mediated Alternative Splicing in the Developing Brain and Dysregulation in Myotonic Dystrophy , 2012, Neuron.

[51]  M. Parmar,et al.  Emery-Dreifuss humeroperoneal muscular dystrophy: cardiac manifestations. , 2012, The Canadian journal of cardiology.

[52]  M. Oshimura,et al.  Transplantation of Genetically Corrected Human iPSC-Derived Progenitors in Mice with Limb-Girdle Muscular Dystrophy , 2012, Science Translational Medicine.

[53]  M. Kyba,et al.  Human ES- and iPS-derived myogenic progenitors restore DYSTROPHIN and improve contractility upon transplantation in dystrophic mice. , 2012, Cell stem cell.

[54]  R. Schugar,et al.  Human muscle-derived cell populations isolated by differential adhesion rates: phenotype and contribution to skeletal muscle regeneration in Mdx/SCID mice. , 2012, Tissue engineering. Part A.

[55]  T. Cooper,et al.  Myotonic dystrophy mouse models: towards rational therapy development. , 2011, Trends in molecular medicine.

[56]  J. Sarsero,et al.  Generation of Induced Pluripotent Stem Cell Lines from Friedreich Ataxia Patients , 2011, Stem Cell Reviews and Reports.

[57]  L. Studer,et al.  Modelling familial dysautonomia in human induced pluripotent stem cells , 2011, Philosophical Transactions of the Royal Society B: Biological Sciences.

[58]  L. Buée,et al.  Mis-splicing of Tau exon 10 in myotonic dystrophy type 1 is reproduced by overexpression of CELF2 but not by MBNL1 silencing. , 2011, Biochimica et biophysica acta.

[59]  Yang Xu,et al.  Immunogenicity of induced pluripotent stem cells , 2011, Nature.

[60]  D. Krainc,et al.  Mitochondrial Parkin Recruitment Is Impaired in Neurons Derived from Mutant PINK1 Induced Pluripotent Stem Cells , 2011, The Journal of Neuroscience.

[61]  N. Benvenisty,et al.  The tumorigenicity of human embryonic and induced pluripotent stem cells , 2011, Nature Reviews Cancer.

[62]  F. Muntoni,et al.  Contribution of Human Muscle-Derived Cells to Skeletal Muscle Regeneration in Dystrophic Host Mice , 2011, PloS one.

[63]  S. Delp,et al.  Short Telomeres and Stem Cell Exhaustion Model Duchenne Muscular Dystrophy in mdx/mTR Mice , 2010, Cell.

[64]  Daniel G. Miller,et al.  Facioscapulohumeral Dystrophy: Incomplete Suppression of a Retrotransposed Gene , 2010, PLoS genetics.

[65]  E. McNally,et al.  Cardiac Assessment in Duchenne and Becker Muscular Dystrophies , 2010, Current heart failure reports.

[66]  S. Thrun,et al.  Substrate Elasticity Regulates Skeletal Muscle Stem Cell Self-Renewal in Culture , 2010, Science.

[67]  R. Jaenisch,et al.  Differentiated Parkinson patient-derived induced pluripotent stem cells grow in the adult rodent brain and reduce motor asymmetry in Parkinsonian rats , 2010, Proceedings of the National Academy of Sciences.

[68]  C. Angelini,et al.  Normal myogenesis and increased apoptosis in myotonic dystrophy type-1 muscle cells , 2010, Cell Death and Differentiation.

[69]  C. Angelini,et al.  Brain involvement in myotonic dystrophies: neuroimaging and neuropsychological comparative study in DM1 and DM2 , 2010, Journal of Neurology.

[70]  S. Matecki,et al.  Leaky RyR2 trigger ventricular arrhythmias in Duchenne muscular dystrophy , 2010, Proceedings of the National Academy of Sciences.

[71]  G. Cossu,et al.  Repairing skeletal muscle: regenerative potential of skeletal muscle stem cells. , 2010, The Journal of clinical investigation.

[72]  M. Oshimura,et al.  Complete Genetic Correction of iPS Cells From Duchenne Muscular Dystrophy , 2009, Molecular therapy : the journal of the American Society of Gene Therapy.

[73]  T. Cooper,et al.  Pathogenic mechanisms of myotonic dystrophy. , 2009, Biochemical Society transactions.

[74]  G. Butler-Browne,et al.  Satellite cell dysfunction contributes to the progressive muscle atrophy in myotonic dystrophy type 1 , 2009, Neuropathology and applied neurobiology.

[75]  Lior Gepstein,et al.  Cardiomyocyte Differentiation of Human Induced Pluripotent Stem Cells , 2009, Circulation.

[76]  A. Viale,et al.  Modeling Pathogenesis and Treatment of Familial Dysautonomia using Patient Specific iPSCs , 2009, Nature.

[77]  J. Emparanza,et al.  Cognitive/personality pattern and triplet expansion size in adult myotonic dystrophy type 1 (DM1): CTG repeats, cognition and personality in DM1 , 2009, Psychological Medicine.

[78]  D. Cohen,et al.  Psychiatric and cognitive phenotype in children and adolescents with myotonic dystrophy , 2009, European Child & Adolescent Psychiatry.

[79]  M. Rudnicki,et al.  Wnt7a activates the planar cell polarity pathway to drive the symmetric expansion of satellite stem cells. , 2009, Cell stem cell.

[80]  Rudolf Jaenisch,et al.  Parkinson's Disease Patient-Derived Induced Pluripotent Stem Cells Free of Viral Reprogramming Factors , 2009, Cell.

[81]  James A. Thomson,et al.  Induced pluripotent stem cells from a spinal muscular atrophy patient , 2009, Nature.

[82]  R. Frants,et al.  Epigenetic mechanisms of facioscapulohumeral muscular dystrophy. , 2008, Mutation research.

[83]  George Q. Daley,et al.  Disease-Specific Induced Pluripotent Stem Cells , 2008, Cell.

[84]  L. Samuelsson,et al.  Autism spectrum conditons in myotonic dystrophy type 1: A study on 57 individuals with congenital and childhood forms , 2008, American journal of medical genetics. Part B, Neuropsychiatric genetics : the official publication of the International Society of Psychiatric Genetics.

[85]  Hynek Wichterle,et al.  Induced Pluripotent Stem Cells Generated from Patients with ALS Can Be Differentiated into Motor Neurons , 2008, Science.

[86]  J. Odorico,et al.  Alternative sources of pluripotency: science, ethics, and stem cells. , 2008, Transplantation reviews.

[87]  T. Cooper,et al.  Overexpression of MBNL1 fetal isoforms and modified splicing of Tau in the DM1 brain: Two individual consequences of CUG trinucleotide repeats , 2008, Experimental Neurology.

[88]  Shulan Tian,et al.  Induced Pluripotent Stem Cell Lines Derived from Human Somatic Cells , 2007, Science.

[89]  T. Ichisaka,et al.  Induction of Pluripotent Stem Cells from Adult Human Fibroblasts by Defined Factors , 2007, Cell.

[90]  Marius Wernig,et al.  Direct reprogramming of genetically unmodified fibroblasts into pluripotent stem cells , 2007, Nature Biotechnology.

[91]  M. Rudnicki,et al.  Stem cell based therapies to treat muscular dystrophy. , 2007, Biochimica et biophysica acta.

[92]  D. Allen,et al.  Intracellular calcium handling in ventricular myocytes from mdx mice. , 2007, American journal of physiology. Heart and circulatory physiology.

[93]  A. Pestronk,et al.  Limb-Girdle Muscular Dystrophy in the United States , 2006, Journal of neuropathology and experimental neurology.

[94]  A. Delacourte,et al.  ETR‐3 represses Tau exons 2/3 inclusion, a splicing event abnormally enhanced in myotonic dystrophy type I , 2006, Journal of neuroscience research.

[95]  S. Yamanaka,et al.  Induction of Pluripotent Stem Cells from Mouse Embryonic and Adult Fibroblast Cultures by Defined Factors , 2006, Cell.

[96]  B. Wong,et al.  Abnormalities of diastolic function precede dilated cardiomyopathy associated with Duchenne muscular dystrophy. , 2006, Journal of the American Society of Echocardiography : official publication of the American Society of Echocardiography.

[97]  T. Cooper,et al.  RNA-mediated neuromuscular disorders. , 2006, Annual review of neuroscience.

[98]  T. Cooper,et al.  Brain-specific change in alternative splicing of Tau exon 6 in myotonic dystrophy type 1. , 2006, Biochimica et biophysica acta.

[99]  A. Delacourte,et al.  Similar brain tau pathology in DM2/PROMM and DM1/Steinert disease , 2005, Neurology.

[100]  Charlotte Collins,et al.  Direct Isolation of Satellite Cells for Skeletal Muscle Regeneration , 2005, Science.

[101]  S. Day,et al.  Dystrophic heart failure blocked by membrane sealant poloxamer , 2005, Nature.

[102]  M. Swanson,et al.  Myotonic dystrophy type 1 is associated with nuclear foci of mutant RNA, sequestration of muscleblind proteins and deregulated alternative splicing in neurons. , 2004, Human molecular genetics.

[103]  J. Bourke,et al.  Cardiac and respiratory failure in limb‐girdle muscular dystrophy 2I , 2004, Annals of neurology.

[104]  M. Vorgerd,et al.  FKRP (826C>A) frequently causes limb-girdle muscular dystrophy in German patients , 2004, Journal of Medical Genetics.

[105]  G. Doucet,et al.  Viral vector producing antisense RNA restores myotonic dystrophy myoblast functions , 2003, Gene Therapy.

[106]  Mark A. Kay,et al.  Progress and problems with the use of viral vectors for gene therapy , 2003, Nature Reviews Genetics.

[107]  J. Bourke,et al.  The phenotype of limb-girdle muscular dystrophy type 2I , 2003, Neurology.

[108]  Susan C. Brown,et al.  Phenotypic spectrum associated with mutations in the fukutin‐related protein gene , 2003, Annals of neurology.

[109]  J. Puymirat,et al.  Decreased levels of myotonic dystrophy protein kinase (DMPK) and delayed differentiation in human myotonic dystrophy myoblasts , 2001, Neuromuscular Disorders.

[110]  L. Timchenko,et al.  Molecular Basis for Impaired Muscle Differentiation in Myotonic Dystrophy , 2001, Molecular and Cellular Biology.

[111]  C. Junien,et al.  Defective satellite cells in congenital myotonic dystrophy. , 2001, Human molecular genetics.

[112]  A. Delacourte,et al.  Dysregulation of human brain microtubule-associated tau mRNA maturation in myotonic dystrophy type 1. , 2001, Human molecular genetics.

[113]  K. Campbell,et al.  Animal models for muscular dystrophy: valuable tools for the development of therapies. , 2000, Human molecular genetics.

[114]  J. Morgan,et al.  Evidence for a myogenic stem cell that is exhausted in dystrophic muscle. , 2000, Journal of cell science.

[115]  S. Terae,et al.  Anterior temporal white matter lesions in myotonic dystrophy with intellectual impairment: an MRI and neuropathological study , 1998, Neuroradiology.

[116]  J. Rommens,et al.  Short GCG expansions in the PABP2 gene cause oculopharyngeal muscular dystrophy , 1998, Nature Genetics.

[117]  J. Steyaert,et al.  A study of the cognitive and psychological profile in 16 children with congenital or juvenile myotonic dystrophy , 1997, Clinical genetics.

[118]  N. Ueno,et al.  Mesodermal subdivision along the mediolateral axis in chicken controlled by different concentrations of BMP-4. , 1997, Development.

[119]  P. McKenna,et al.  Mild myotonic dystrophy is associated with memory impairment in the context of normal general intelligence. , 1997, Journal of medical genetics.

[120]  A. Delacourte,et al.  Specific tau variants in the brains of patients with myotonic dystrophy , 1996, Neurology.

[121]  R. Moon,et al.  The axis-inducing activity, stability, and subcellular distribution of beta-catenin is regulated in Xenopus embryos by glycogen synthase kinase 3. , 1996, Genes & development.

[122]  R. R. Rice,et al.  Myoblast transfer in the treatment of Duchenne's muscular dystrophy. , 1995, The New England journal of medicine.

[123]  M. Gebbia,et al.  Lack of mRNA and dystrophin expression in DMD patients three months after myoblast transfer , 1995, Neuromuscular Disorders.

[124]  M. Roig,et al.  Presentation, clinical course, and outcome of the congenital form of myotonic dystrophy. , 1994, Pediatric neurology.

[125]  George Karpati,et al.  Myoblast transfer in duchenne muscular dystrophy , 1993, Annals of neurology.

[126]  J. Bouchard,et al.  Human myoblast transplantation: Preliminary results of 4 cases , 1992, Muscle & nerve.

[127]  H. Blau,et al.  Normal dystrophin transcripts detected in Duchenne muscular dystrophy patients after myoblast transplantation , 1992, Nature.

[128]  M. Matsunaga,et al.  Topography of Alzheimer's neurofibrillary change distribution in myotonic dystrophy. , 1990, Clinical neuropathology.

[129]  T. Bertorini,et al.  Dystrophin production induced by myoblast transfer therapy in Duchenne muscular dystrophy , 1990, The Lancet.

[130]  S. Mitake,et al.  [Development of Alzheimer neurofibrillary changes in two autopsy cases of myotonic dystrophy]. , 1989, Rinsho shinkeigaku = Clinical neurology.

[131]  J. Barbet,et al.  Immaturity of muscle fibers in the congenital form of myotonic dystrophy: Its consequences and its origin , 1988, Journal of the Neurological Sciences.

[132]  T. Partridge,et al.  THE mdx MOUSE SKELETAL MUSCLE MYOPATHY: I. A HISTOLOGICAL, MORPHOMETRIC AND BIOCHEMICAL INVESTIGATION , 1988, Neuropathology and applied neurobiology.

[133]  D. Shotton,et al.  Muscular dystrophy in the mdx mouse: Histopathology of the soleus and extensor digitorum longus muscles , 1987, Journal of the Neurological Sciences.

[134]  M. Duveneck,et al.  Intellectual and cognitive function in adults with myotonic muscular dystrophy. , 1986, Archives of physical medicine and rehabilitation.

[135]  T. Bird,et al.  Cognitive and personality function in myotonic muscular dystrophy. , 1983, Journal of neurology, neurosurgery, and psychiatry.

[136]  V. Sahgal,et al.  Skeletal muscle in preterm infants with congenital myotonic dystrophy Morphologic and histochemical study , 1983, Journal of the Neurological Sciences.

[137]  L. Elsas,et al.  Emery‐dreifuss humeroperoneal muscular dystorphy: An X‐linked myopathy with unusual contractures and bradycardia , 1981, Annals of neurology.

[138]  H. Sarnat,et al.  Maturational arrest of fetal muscle in neonatal myotonic dystrophy. A pathologic study of four cases. , 1976, Archives of neurology.

[139]  K. Woltjen,et al.  Directed Myogenic Differentiation of Human Induced Pluripotent Stem Cells. , 2016, Methods in molecular biology.

[140]  I. Komuro,et al.  Generation of Induced Pluripotent Stem Cells From Patients With Duchenne Muscular Dystrophy and Their Induction to Cardiomyocytes. , 2016, International heart journal.

[141]  R. Perlingeiro,et al.  Derivation of Skeletal Myogenic Precursors from Human Pluripotent Stem Cells Using Conditional Expression of PAX7. , 2016, Methods in molecular biology.

[142]  M. Ciemerych,et al.  Cell therapy in Duchenne muscular dystrophy treatment: clinical trials overview. , 2015, Critical reviews in eukaryotic gene expression.

[143]  C. Thornton.  Myotonic dystrophy. , 2014, Neurologic clinics.

[144]  V. Romeo.  Myotonic Dystrophy Type 1 or Steinert's disease. , 2012, Advances in experimental medicine and biology.

[145]  K. Sidhu,et al.  Derivation, propagation, and characterization of neuroprogenitors from pluripotent stem cells (hESCs and hiPSCs). , 2012, Methods in molecular biology.

[146]  John K. Hall,et al.  Animal models of muscular dystrophy. , 2012, Progress in molecular biology and translational science.

[147]  T. Ashizawa,et al.  Myotonic dystrophy types 1 and 2. , 2011, Handbook of clinical neurology.

[148]  B. Thiers.  Induction of Pluripotent Stem Cells from Adult Human Fibroblasts by Defined Factors , 2008 .

[149]  J. O'Shea,et al.  Cardiomyopathy of Duchenne muscular dystrophy , 2005, Pediatric Cardiology.

[150]  E. Nanba,et al.  [Myotonic dystrophy]. , 2005, Nihon rinsho. Japanese journal of clinical medicine.

[151]  N. Otsuka,et al.  Presenile appearance of abundant Alzheimer's neurofibrillary tangles without senile plaques in the brain in myotonic dystrophy , 2004, Acta Neuropathologica.

[152]  P. Wollan,et al.  Skeletal, cardiac, and smooth muscle failure in Duchenne muscular dystrophy. , 1996, Pediatric neurology.