scholarly article | Q13442814 |
P2093 | author name string | Naohiro Terada | |
Tetsuo Ashizawa | |||
Guangbin Xia | |||
P2860 | cites work | Facioscapulohumeral dystrophy: incomplete suppression of a retrotransposed gene | Q21563391 |
Leaky RyR2 trigger ventricular arrhythmias in Duchenne muscular dystrophy | Q24295360 | ||
Mitochondrial Parkin recruitment is impaired in neurons derived from mutant PINK1 induced pluripotent stem cells | Q24300095 | ||
Short GCG expansions in the PABP2 gene cause oculopharyngeal muscular dystrophy | Q24308826 | ||
Molecular basis for impaired muscle differentiation in myotonic dystrophy | Q24548314 | ||
Muscleblind-like 2-mediated alternative splicing in the developing brain and dysregulation in myotonic dystrophy | Q24617996 | ||
Substrate elasticity regulates skeletal muscle stem cell self-renewal in culture | Q24623940 | ||
Short telomeres and stem cell exhaustion model Duchenne muscular dystrophy in mdx/mTR mice | Q24633931 | ||
Disease-specific induced pluripotent stem cells | Q24641981 | ||
Myogenic Precursors from iPS Cells for Skeletal Muscle Cell Replacement Therapy | Q26799633 | ||
Early pathogenesis of Duchenne muscular dystrophy modelled in patient-derived human induced pluripotent stem cells. | Q27308743 | ||
Efficient and reproducible myogenic differentiation from human iPS cells: prospects for modeling Miyoshi Myopathy in vitro | Q27324593 | ||
Modeling induced pluripotent stem cells from fibroblasts of Duchenne muscular dystrophy patients | Q44227546 | ||
Viral vector producing antisense RNA restores myotonic dystrophy myoblast functions | Q44409505 | ||
The phenotype of limb-girdle muscular dystrophy type 2I | Q44411539 | ||
Efficient derivation and inducible differentiation of expandable skeletal myogenic cells from human ES and patient-specific iPS cells. | Q45876520 | ||
Normal dystrophin transcripts detected in Duchenne muscular dystrophy patients after myoblast transplantation | Q46050411 | ||
Dystrophic heart failure blocked by membrane sealant poloxamer | Q46604988 | ||
CRISPR/Cas9-Mediated Deletion of CTG Expansions Recovers Normal Phenotype in Myogenic Cells Derived from Myotonic Dystrophy 1 Patients | Q47100283 | ||
Cognitive/personality pattern and triplet expansion size in adult myotonic dystrophy type 1 (DM1): CTG repeats, cognition and personality in DM1. | Q47176340 | ||
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). | Q47649427 | ||
Presentation, clinical course, and outcome of the congenital form of myotonic dystrophy | Q47655568 | ||
Immaturity of muscle fibers in the congenital form of myotonic dystrophy: its consequences and its origin | Q47689534 | ||
Cardiac and respiratory failure in limb-girdle muscular dystrophy 2I. | Q47906360 | ||
Correction of diverse muscular dystrophy mutations in human engineered heart muscle by single-site genome editing. | Q48041681 | ||
Brain involvement in myotonic dystrophies: neuroimaging and neuropsychological comparative study in DM1 and DM2. | Q48276028 | ||
Phenotypic spectrum associated with mutations in the fukutin-related protein gene | Q48350456 | ||
Anterior temporal white matter lesions in myotonic dystrophy with intellectual impairment: an MRI and neuropathological study | Q48398995 | ||
CRISPR/Cas9-Induced (CTG⋅CAG)n Repeat Instability in the Myotonic Dystrophy Type 1 Locus: Implications for Therapeutic Genome Editing. | Q48854625 | ||
Specific tau variants in the brains of patients with myotonic dystrophy | Q48935349 | ||
A human iPS cell myogenic differentiation system permitting high-throughput drug screening | Q50069427 | ||
Autism spectrum conditions in myotonic dystrophy type 1: a study on 57 individuals with congenital and childhood forms | Q50310770 | ||
Genome modification leads to phenotype reversal in human myotonic dystrophy type 1 induced pluripotent stem cell-derived neural stem cells | Q40979450 | ||
Generation of Induced Pluripotent Stem Cells From Patients With Duchenne Muscular Dystrophy and Their Induction to Cardiomyocytes | Q41045809 | ||
Genome Therapy of Myotonic Dystrophy Type 1 iPS Cells for Development of Autologous Stem Cell Therapy | Q41092886 | ||
Myoblasts derived from normal hESCs and dystrophic hiPSCs efficiently fuse with existing muscle fibers following transplantation | Q41136531 | ||
Derivation and expansion of PAX7-positive muscle progenitors from human and mouse embryonic stem cells | Q41823910 | ||
Precise correction of the dystrophin gene in duchenne muscular dystrophy patient induced pluripotent stem cells by TALEN and CRISPR-Cas9. | Q41873090 | ||
Myogenic differentiation of muscular dystrophy-specific induced pluripotent stem cells for use in drug discovery | Q41956611 | ||
Derivation of Skeletal Myogenic Precursors from Human Pluripotent Stem Cells Using Conditional Expression of PAX7. | Q42181288 | ||
Derivation and FACS-mediated purification of PAX3+/PAX7+ skeletal muscle precursors from human pluripotent stem cells | Q42273400 | ||
Myoblast transfer in Duchenne muscular dystrophy | Q42512550 | ||
Direct reprogramming of genetically unmodified fibroblasts into pluripotent stem cells | Q42807434 | ||
Transplantation of genetically corrected human iPSC-derived progenitors in mice with limb-girdle muscular dystrophy. | Q42819955 | ||
Dysregulation of human brain microtubule-associated tau mRNA maturation in myotonic dystrophy type 1. | Q43759003 | ||
Topography of Alzheimer's neurofibrillary change distribution in myotonic dystrophy | Q43921215 | ||
Induced pluripotent stem cell lines derived from human somatic cells | Q27860597 | ||
Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors | Q27860937 | ||
Induction of pluripotent stem cells from adult human fibroblasts by defined factors | Q27860967 | ||
Potential of Induced Pluripotent Stem Cells (iPSCs) for Treating Age-Related Macular Degeneration (AMD) | Q28077285 | ||
Induced pluripotent stem cells in dentistry | Q28078372 | ||
Mesodermal subdivision along the mediolateral axis in chicken controlled by different concentrations of BMP-4 | Q28239666 | ||
Differentiation of pluripotent stem cells to muscle fiber to model Duchenne muscular dystrophy | Q28265827 | ||
Wnt7a activates the planar cell polarity pathway to drive the symmetric expansion of satellite stem cells | Q28511912 | ||
Parkinson's disease patient-derived induced pluripotent stem cells free of viral reprogramming factors | Q28749672 | ||
A POGLUT1 mutation causes a muscular dystrophy with reduced Notch signaling and satellite cell loss. | Q29347528 | ||
Induced pluripotent stem cells from a spinal muscular atrophy patient | Q29614340 | ||
The axis-inducing activity, stability, and subcellular distribution of beta-catenin is regulated in Xenopus embryos by glycogen synthase kinase 3 | Q29614380 | ||
Immunogenicity of induced pluripotent stem cells | Q29616185 | ||
Induced pluripotent stem cells generated from patients with ALS can be differentiated into motor neurons | Q29616199 | ||
Derivation of myogenic progenitors directly from human pluripotent stem cells using a sphere-based culture | Q30577387 | ||
A Novel Protocol for Directed Differentiation of C9orf72-Associated Human Induced Pluripotent Stem Cells Into Contractile Skeletal Myotubes | Q30824665 | ||
Repairing skeletal muscle: regenerative potential of skeletal muscle stem cells | Q33559684 | ||
Mild myotonic dystrophy is associated with memory impairment in the context of normal general intelligence | Q33678560 | ||
Contribution of human muscle-derived cells to skeletal muscle regeneration in dystrophic host mice | Q33847288 | ||
Skeletal muscle generated from induced pluripotent stem cells - induction and application | Q33854768 | ||
Notch signaling deficiency underlies age-dependent depletion of satellite cells in muscular dystrophy. | Q33935640 | ||
Animal models for muscular dystrophy: valuable tools for the development of therapies | Q34045919 | ||
Differentiated Parkinson patient-derived induced pluripotent stem cells grow in the adult rodent brain and reduce motor asymmetry in Parkinsonian rats | Q34115609 | ||
Molecular, physiological, and motor performance defects in DMSXL mice carrying >1,000 CTG repeats from the human DM1 locus | Q34499428 | ||
Autologous Induced Stem-Cell-Derived Retinal Cells for Macular Degeneration. | Q34553328 | ||
Modelling familial dysautonomia in human induced pluripotent stem cells | Q35088526 | ||
Progress and problems with the use of viral vectors for gene therapy | Q35120176 | ||
FKRP (826C>A) frequently causes limb-girdle muscular dystrophy in German patients | Q35445374 | ||
Modeling and study of the mechanism of dilated cardiomyopathy using induced pluripotent stem cells derived from individuals with Duchenne muscular dystrophy | Q35558056 | ||
Human muscle-derived cell populations isolated by differential adhesion rates: phenotype and contribution to skeletal muscle regeneration in Mdx/SCID mice | Q35706322 | ||
Prevalence of Duchenne and Becker muscular dystrophies in the United States | Q35770545 | ||
Human ES- and iPS-derived myogenic progenitors restore DYSTROPHIN and improve contractility upon transplantation in dystrophic mice. | Q35947481 | ||
Human Satellite Cell Transplantation and Regeneration from Diverse Skeletal Muscles | Q36201566 | ||
Overexpression of MBNL1 fetal isoforms and modified splicing of Tau in the DM1 brain: two individual consequences of CUG trinucleotide repeats. | Q50651329 | ||
Direct isolation of satellite cells for skeletal muscle regeneration. | Q50756094 | ||
A New Mouse Model of Limb-Girdle Muscular Dystrophy Type 2I Homozygous for the Common L276I Mutation Mimicking the Mild Phenotype in Humans. | Q50911860 | ||
A study of the cognitive and psychological profile in 16 children with congenital or juvenile myotonic dystrophy. | Q51100898 | ||
Satellite cell dysfunction contributes to the progressive muscle atrophy in myotonic dystrophy type 1. | Q51829611 | ||
Intellectual and cognitive function in adults with myotonic muscular dystrophy. | Q52140183 | ||
Evidence for a myogenic stem cell that is exhausted in dystrophic muscle. | Q52168124 | ||
Muscular dystrophy in the mdx mouse: histopathology of the soleus and extensor digitorum longus muscles. | Q52255659 | ||
[Development of Alzheimer neurofibrillary changes in two autopsy cases of myotonic dystrophy] | Q53188055 | ||
Presenile appearance of abundant Alzheimer's neurofibrillary tangles without senile plaques in the brain in myotonic dystrophy. | Q53296761 | ||
Directed Myogenic Differentiation of Human Induced Pluripotent Stem Cells. | Q53506908 | ||
At the Crossroads of Clinical and Preclinical Research for Muscular Dystrophy-Are We Closer to Effective Treatment for Patients? | Q55361250 | ||
Normal myogenesis and increased apoptosis in myotonic dystrophy type-1 muscle cells | Q58923911 | ||
Lack of mRNA and dystrophin expression in DMD patients three months after myoblast transfer | Q61828091 | ||
Dystrophin production induced by myoblast transfer therapy in Duchenne muscular dystrophy | Q67668959 | ||
Cardiomyopathy of Duchenne muscular dystrophy | Q68052472 | ||
The mdx mouse skeletal muscle myopathy: I. A histological, morphometric and biochemical investigation | Q70054992 | ||
Emery‐dreifuss humeroperoneal muscular dystorphy: An X‐linked myopathy with unusual contractures and bradycardia | Q70965803 | ||
Skeletal, cardiac, and smooth muscle failure in Duchenne muscular dystrophy | Q71127157 | ||
Myoblast Transfer in the Treatment of Duchenne's Muscular Dystrophy | Q72000232 | ||
Skeletal muscle in preterm infants with congenital myotonic dystrophy. Morphologic and histochemical study | Q72785527 | ||
Defective satellite cells in congenital myotonic dystrophy | Q74599300 | ||
Decreased levels of myotonic dystrophy protein kinase (DMPK) and delayed differentiation in human myotonic dystrophy myoblasts | Q74609017 | ||
Intracellular calcium handling in ventricular myocytes from mdx mice | Q79166127 | ||
Limb-girdle muscular dystrophy in the United States | Q79189595 | ||
Abnormalities of diastolic function precede dilated cardiomyopathy associated with Duchenne muscular dystrophy | Q79850222 | ||
Myotonic dystrophy type 1 is associated with nuclear foci of mutant RNA, sequestration of muscleblind proteins and deregulated alternative splicing in neurons | Q80889062 | ||
Similar brain tau pathology in DM2/PROMM and DM1/Steinert disease | Q81511866 | ||
Emery-Dreifuss humeroperoneal muscular dystrophy: cardiac manifestations | Q83821658 | ||
Derivation, propagation, and characterization of neuroprogenitors from pluripotent stem cells (hESCs and hiPSCs) | Q83974067 | ||
Myotonic dystrophy | Q87411199 | ||
Ion Channel Dysfunctions in Dilated Cardiomyopathy in Limb-Girdle Muscular Dystrophy | Q88073295 | ||
Mechanisms of skeletal muscle wasting in a mouse model for myotonic dystrophy type 1 | Q88712420 | ||
Ex Vivo Expansion and In Vivo Self-Renewal of Human Muscle Stem Cells. | Q36221133 | ||
RNA-mediated neuromuscular disorders | Q36507582 | ||
Stem cell based therapies to treat muscular dystrophy | Q36621005 | ||
Generation of neural cells from DM1 induced pluripotent stem cells as cellular model for the study of central nervous system neuropathogenesis | Q36740042 | ||
A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells | Q36782735 | ||
Animal models of muscular dystrophy | Q36915922 | ||
Precise Correction of Disease Mutations in Induced Pluripotent Stem Cells Derived From Patients With Limb Girdle Muscular Dystrophy | Q36951286 | ||
Cognitive and personality function in myotonic muscular dystrophy | Q37029429 | ||
Epigenetic mechanisms of facioscapulohumeral muscular dystrophy | Q37114491 | ||
Alternative sources of pluripotency: science, ethics, and stem cells | Q37218176 | ||
Pathogenic mechanisms of myotonic dystrophy | Q37414636 | ||
Myotonic dystrophy mouse models: towards rational therapy development. | Q37434343 | ||
Modelling pathogenesis and treatment of familial dysautonomia using patient-specific iPSCs. | Q37444336 | ||
Psychiatric and cognitive phenotype in children and adolescents with myotonic dystrophy | Q37526565 | ||
Induced Pluripotent Stem Cells for Traumatic Spinal Cord Injury | Q37592612 | ||
Cardiac Assessment in Duchenne and Becker Muscular Dystrophies | Q37790640 | ||
The tumorigenicity of human embryonic and induced pluripotent stem cells | Q37851350 | ||
Myotonic dystrophy types 1 and 2. | Q37865708 | ||
Myotonic Dystrophy Type 1 or Steinert's disease | Q37992909 | ||
The myotonic dystrophies: molecular, clinical, and therapeutic challenges | Q38045121 | ||
Concise review: stem cell therapy for muscular dystrophies | Q38063847 | ||
Myotonic dystrophy: diagnosis, management and new therapies | Q38239745 | ||
Mis-splicing of Tau exon 10 in myotonic dystrophy type 1 is reproduced by overexpression of CELF2 but not by MBNL1 silencing. | Q38336368 | ||
Cell therapy in Duchenne muscular dystrophy treatment: clinical trials overview. | Q38467615 | ||
iPS cell technologies and their prospect for bone regeneration and disease modeling: A mini review | Q38733804 | ||
Generation of human muscle fibers and satellite-like cells from human pluripotent stem cells in vitro | Q38748704 | ||
A Human Pluripotent Stem Cell Model of Facioscapulohumeral Muscular Dystrophy-Affected Skeletal Muscles. | Q38768975 | ||
Clinical Application of Pluripotent Stem Cells: An Alternative Cell-Based Therapy for Treating Liver Diseases? | Q38919880 | ||
Clinical potential of human-induced pluripotent stem cells : Perspectives of induced pluripotent stem cells | Q39024182 | ||
Cellular Reprogramming, Genome Editing, and Alternative CRISPR Cas9 Technologies for Precise Gene Therapy of Duchenne Muscular Dystrophy | Q39368114 | ||
Generation of induced pluripotent stem cell lines from Friedreich ataxia patients. | Q39616684 | ||
Complete genetic correction of ips cells from Duchenne muscular dystrophy | Q39641162 | ||
Maturational arrest of fetal muscle in neonatal myotonic dystrophy. A pathologic study of four cases | Q39774611 | ||
Cardiomyocyte differentiation of human induced pluripotent stem cells | Q39794134 | ||
ETR-3 represses Tau exons 2/3 inclusion, a splicing event abnormally enhanced in myotonic dystrophy type I. | Q40252149 | ||
Brain-specific change in alternative splicing of Tau exon 6 in myotonic dystrophy type 1. | Q40314719 | ||
Human myoblast transplantation: preliminary results of 4 cases | Q40734292 | ||
P275 | copyright license | Creative Commons Attribution 4.0 International | Q20007257 |
P6216 | copyright status | copyrighted | Q50423863 |
P433 | issue | 4 | |
P921 | main subject | orphan disease | Q15712664 |
P304 | page(s) | 299-309 | |
P577 | publication date | 2018-10-04 | |
P1433 | published in | Current stem cell reports | Q27726279 |
P1476 | title | Human iPSC Models to Study Orphan Diseases: Muscular Dystrophies | |
P478 | volume | 4 |
Q92258204 | CRISPR/Cas Applications in Myotonic Dystrophy: Expanding Opportunities | cites work | P2860 |
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