4.7 Article

Genome Modification Leads to Phenotype Reversal in Human Myotonic Dystrophy Type 1 Induced Pluripotent Stem Cell-Derived Neural Stem Cells

相关参考文献

注意:仅列出部分参考文献,下载原文获取全部文献信息。
Article Biochemistry & Molecular Biology

Mechanisms of transcriptional dysregulation in repeat expansion disorders

Matthias Groh et al.

BIOCHEMICAL SOCIETY TRANSACTIONS (2014)

Review Neurosciences

The past, present and future of stem cell clinical trials for ALS

Gretchen M. Thomsen et al.

EXPERIMENTAL NEUROLOGY (2014)

Review Chemistry, Multidisciplinary

Mesenchymal Stem Cell Therapy for Neurodegenerative Diseases

Lukui Chen et al.

JOURNAL OF NANOSCIENCE AND NANOTECHNOLOGY (2014)

Article Medicine, Research & Experimental

Alzheimer's Disease and Stem Cell Therapy

Sung S. Choi et al.

Experimental Neurobiology (2014)

Review Clinical Neurology

Cell Therapy Strategies vs. Paracrine Effect in Huntington's Disease

Wooseok Im et al.

JOURNAL OF MOVEMENT DISORDERS (2014)

Article Genetics & Heredity

Bidirectional transcription of trinucleotide repeats: Roles for excision repair

Helen Budworth et al.

DNA REPAIR (2013)

Review Biochemistry & Molecular Biology

Repeat-associated non-ATG (RAN) translation in neurological disease

John D. Cleary et al.

HUMAN MOLECULAR GENETICS (2013)

Article Clinical Neurology

Stem cell grafting in parkinsonism – Why, how and when

Johannes P.J.M. de Munter et al.

PARKINSONISM & RELATED DISORDERS (2013)

Article Medicine, Research & Experimental

Synaptic protein dysregulation in myotonic dystrophy type 1 Disease neuropathogenesis beyond missplicing

Oscar Hernandez-Hernandez et al.

RARE DISEASES (2013)

Article Biochemistry & Molecular Biology

Rapid and Cost-Effective Gene Targeting in Rat Embryonic Stem Cells by TALENs

Chang Tong et al.

JOURNAL OF GENETICS AND GENOMICS (2012)

Review Genetics & Heredity

Human induced pluripotent stem cells-from mechanisms to clinical applications

Katharina Drews et al.

JOURNAL OF MOLECULAR MEDICINE-JMM (2012)

Article Multidisciplinary Sciences

Targeting nuclear RNA for in vivo correction of myotonic dystrophy

Thurman M. Wheeler et al.

NATURE (2012)

Article Biochemistry & Molecular Biology

Highly efficient generation of heritable zebrafish gene mutations using homo- and heterodimeric TALENs

Lindsay Cade et al.

NUCLEIC ACIDS RESEARCH (2012)

Article Multidisciplinary Sciences

RNase H-mediated degradation of toxic RNA in myotonic dystrophy type 1

Johanna E. Lee et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2012)

Article Biochemistry & Molecular Biology

Mis-splicing of Tau exon 10 in myotonic dystrophy type 1 is reproduced by overexpression of CELF2 but not by MBNL1 silencing

C. M. Dhaenens et al.

BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE (2011)

Review Biochemistry & Molecular Biology

Gain of RNA function in pathological cases: Focus on myotonic dystrophy

A. F. Klein et al.

BIOCHIMIE (2011)

Article Biotechnology & Applied Microbiology

A TALE nuclease architecture for efficient genome editing

Jeffrey C. Miller et al.

NATURE BIOTECHNOLOGY (2011)

Article Biotechnology & Applied Microbiology

Genetic engineering of human pluripotent cells using TALE nucleases

Dirk Hockemeyer et al.

NATURE BIOTECHNOLOGY (2011)

Letter Biotechnology & Applied Microbiology

Targeted gene disruption in somatic zebrafish cells using engineered TALENs

Jeffry D. Sander et al.

NATURE BIOTECHNOLOGY (2011)

Article Biotechnology & Applied Microbiology

A functionally characterized test set of human induced pluripotent stem cells

Gabriella L. Boulting et al.

NATURE BIOTECHNOLOGY (2011)

Article Biochemistry & Molecular Biology

TAL nucleases (TALNs): hybrid proteins composed of TAL effectors and FokI DNA-cleavage domain

Ting Li et al.

NUCLEIC ACIDS RESEARCH (2011)

Article Biochemistry & Molecular Biology

A novel TALE nuclease scaffold enables high genome editing activity in combination with low toxicity

Claudio Mussolino et al.

NUCLEIC ACIDS RESEARCH (2011)

Article Multidisciplinary Sciences

Non-ATG-initiated translation directed by microsatellite expansions

Tao Zu et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2011)

Review Biochemistry & Molecular Biology

Myotonic dystrophy mouse models: towards rational therapy development

Mario Gomes-Pereira et al.

TRENDS IN MOLECULAR MEDICINE (2011)

Article Clinical Neurology

Introduction to Induced Pluripotent Stem Cells: Advancing the Potential for Personalized Medicine

Johnathan D. Ebben et al.

WORLD NEUROSURGERY (2011)

Article Multidisciplinary Sciences

iPS cell technology in regenerative medicine

Christopher J. Lengner

SKELETAL BIOLOGY AND MEDICINE (2010)

Article Biochemistry & Molecular Biology

Pathogenic mechanisms of myotonic dystrophy

Johanna E. Lee et al.

BIOCHEMICAL SOCIETY TRANSACTIONS (2009)

Review Psychology, Developmental

Psychiatric and cognitive phenotype in children and adolescents with myotonic dystrophy

Marie Douniol et al.

EUROPEAN CHILD & ADOLESCENT PSYCHIATRY (2009)

Review Biochemistry & Molecular Biology

Mechanisms of RNA-mediated Disease

Jason R. O'Rourke et al.

JOURNAL OF BIOLOGICAL CHEMISTRY (2009)

Article Multidisciplinary Sciences

Feeder-free derivation of induced pluripotent stem cells from adult human adipose stem cells

Ning Sun et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2009)

Article Multidisciplinary Sciences

Pentamidine reverses the splicing defects associated with myotonic dystrophy

M. Bryan Warf et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2009)

Article Multidisciplinary Sciences

Triplet-repeat oligonucleotide-mediated reversal of RNA toxicity in myotonic dystrophy

Susan A. M. Mulders et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2009)

Article Multidisciplinary Sciences

Reversal of RNA Dominance by Displacement of Protein Sequestered on Triplet Repeat RNA

Thurman M. Wheeler et al.

SCIENCE (2009)

Article Genetics & Heredity

Autism spectrum conditons in myotonic dystrophy type 1:: A study on 57 individuals with congenital and childhood forms

Anne-Berit Ekstrom et al.

AMERICAN JOURNAL OF MEDICAL GENETICS PART B-NEUROPSYCHIATRIC GENETICS (2008)

Article Biotechnology & Applied Microbiology

Efficient and rapid generation of induced pluripotent stem cells from human keratinocytes

Trond Aasen et al.

NATURE BIOTECHNOLOGY (2008)

Article Multidisciplinary Sciences

Expanded CTG repeats within the DMPK 3′ UTR causes severe skeletal muscle wasting in an inducible mouse model for myotonic dystrophy

James P. Orengo et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2008)

Article Biochemistry & Molecular Biology

Early evolution of histone mRNA 39 end processing

Marcela Davila Lopez et al.

Article Biochemistry & Molecular Biology

Induction of pluripotent stem cells from adult human fibroblasts by defined factors

Kazutoshi Takahashi et al.

Article Biotechnology & Applied Microbiology

Direct reprogramming of genetically unmodified fibroblasts into pluripotent stem cells

Alexander Meissner et al.

NATURE BIOTECHNOLOGY (2007)

Article Biochemistry & Molecular Biology

Systematic variation in mRNA 3 '-processing signals during mouse spermatogenesis

Donglin Liu et al.

NUCLEIC ACIDS RESEARCH (2007)

Article Clinical Neurology

Similar brain tau pathology in DM2/PROMM and DM1/Steinert disease

CA Maurage et al.

NEUROLOGY (2005)

Article Biochemistry & Molecular Biology

Antisense transcription and heterochromatin at the DM1 CTG repeats are constrained by CTCF

DH Cho et al.

MOLECULAR CELL (2005)

Article Biochemistry & Molecular Biology

Alternative splicing controls myotonic dystrophy protein kinase structure, enzymatic activity, and subcellular localization

DG Wansink et al.

MOLECULAR AND CELLULAR BIOLOGY (2003)

Article Biotechnology & Applied Microbiology

Hammerhead ribozyme-mediated destruction of nuclear foci in myotonic dystrophy myoblasts

MA Langlois et al.

MOLECULAR THERAPY (2003)

Article Multidisciplinary Sciences

Myotonic dystrophy in transgenic mice expressing an expanded CUG repeat

A Mankodi et al.

SCIENCE (2000)