Human Pluripotent Stem Cells Modeling Neurodegenerative Diseases

Modeling of human neurodegenerative diseases in animals has led to important advances in the understanding of pathogenic mechanisms and has opened avenues for curative ap‐ proaches. However, inherent genetic, developmental and anatomical species differences between humans and animals frequently resulted in imperfect phenotypic correlations between animal models and human diseases. This might account for the observed hampered translation of promising preclinical treatment studies in animal models towards clinics.

• Publication year

  2013

• Venue

  Unknown venue

• Publication date

  2013-08-28

• Fields of study

  Biology, Medicine

• Identifiers
  DOI 10.5772/55772
• External record

  Open on Semantic Scholar

• Source metadata

  Semantic Scholar

• No claims are published for this paper.

• No concepts are published for this paper.

• The Neuropathology of Huntington's Disease.

  2015cited by this paper
• Supplementary Materials and Methods

  2013cited by this paper
• The first reported generation of several induced pluripotent stem cell lines from homozygous and heterozygous Huntington's disease patients demonstrates mutation related enhanced lysosomal activity.

  2012cited by this paper
• Efficient Generation of A9 Midbrain Dopaminergic Neurons by Lentiviral Delivery of LMX1A in Human Embryonic Stem Cells and Induced Pluripotent Stem Cells

  2012cited by this paper
• Directed Induction of Functional Motor Neuron-Like Cells from Genetically Engineered Human Mesenchymal Stem Cells

  2012cited by this paper
• Accelerated generation of human induced pluripotent stem cells with retroviral transduction and chemical inhibitors under physiological hypoxia.

  2012cited by this paper
• Generating GABAergic cerebral cortical interneurons from mouse and human embryonic stem cells.

  2012cited by this paper
• Inhibition of Apoptosis Blocks Human Motor Neuron Cell Death in a Stem Cell Model of Spinal Muscular Atrophy

  2012influential reference
• Probing sporadic and familial Alzheimer’s disease using induced pluripotent stem cells

  2012cited by this paper
• Activin induces cortical interneuron identity and differentiation in embryonic stem cell-derived telencephalic neural precursors

  2012cited by this paper
• Understanding the genetic and molecular pathogenesis of Friedreich’s ataxia through animal and cellular models

  2012cited by this paper
• Pharmacological Modulation of the Hedgehog Pathway Differentially Affects Dorsal/Ventral Patterning in Mouse and Human Embryonic Stem Cell Models of Telencephalic Development

  2012cited by this paper
• Directed differentiation of motor neuron cell-like cells from human adipose-derived stem cells in vitro

  2011cited by this paper
• Retinoid-independent motor neurogenesis from human embryonic stem cells reveals a medial columnar ground state

  2011cited by this paper
• Modelling familial dysautonomia in human induced pluripotent stem cells

  2011cited by this paper
• Generation of Induced Pluripotent Stem Cell Lines from Friedreich Ataxia Patients

  2011cited by this paper
• Drosophila FMRP regulates microtubule network formation and axonal transport of mitochondria.

  2011cited by this paper
• Constructing and deconstructing stem cell models of neurological disease.

  2011cited by this paper
• Direct conversion of human fibroblasts to dopaminergic neurons

  2011cited by this paper
• The Controlled Generation of Functional Basal Forebrain Cholinergic Neurons from Human Embryonic Stem Cells

  2011cited by this paper
• Genetic Rodent Models of Amyotrophic Lateral Sclerosis

  2011cited by this paper
• Wnt signaling and a Smad pathway blockade direct the differentiation of human pluripotent stem cells to multipotent neural crest cells

  2011cited by this paper
• Huntington's disease: from molecular pathogenesis to clinical treatment

  2011cited by this paper
• Derivation of Huntington's Disease-Affected Human Embryonic Stem Cell Lines

  2011cited by this paper
• Parkinson's disease induced pluripotent stem cells with triplication of the α-synuclein locus

  2011cited by this paper
• A functionally characterized test set of human induced pluripotent stem cells

  2011cited by this paper
• Directed conversion of Alzheimer's disease patient skin fibroblasts into functional neurons.

  2011cited by this paper
• Methods for making induced pluripotent stem cells: reprogramming à la carte

  2011cited by this paper
• Friedreich's ataxia: pathology, pathogenesis, and molecular genetics.

  2011cited by this paper
• Modeling familial Alzheimer's disease with induced pluripotent stem cells.

  2011cited by this paper
• Induction of human neuronal cells by defined transcription factors

  2011cited by this paper
• Deletion of Exon 20 of the Familial Dysautonomia Gene Ikbkap in Mice Causes Developmental Delay, Cardiovascular Defects, and Early Embryonic Lethality

  2011cited by this paper
• LRRK2 mutant iPSC-derived DA neurons demonstrate increased susceptibility to oxidative stress.

  2011cited by this paper
• SNCA Triplication Parkinson's Patient's iPSC-derived DA Neurons Accumulate α-Synuclein and Are Susceptible to Oxidative Stress

  2011cited by this paper
• Direct generation of functional dopaminergic neurons from mouse and human fibroblasts

  2011cited by this paper
• Pluripotent Stem Cells for the Study of CNS Development

  2011cited by this paper
• Highly efficient miRNA-mediated reprogramming of mouse and human somatic cells to pluripotency.

  2011cited by this paper
• Subregional Specification of Embryonic Stem Cell-Derived Ventral Telencephalic Tissues by Timed and Combinatory Treatment with Extrinsic Signals

  2011cited by this paper
• Amyloid-β and tau — a toxic pas de deux in Alzheimer's disease

  2011cited by this paper
• Modeling human neurodegenerative diseases in transgenic systems

  2011cited by this paper
• Reprogramming of mouse and human cells to pluripotency using mature microRNAs.

  2011cited by this paper
• Mitochondrial Parkin Recruitment Is Impaired in Neurons Derived from Mutant PINK1 Induced Pluripotent Stem Cells

  2011cited by this paper
• A Targeted NKX2.1 Human Embryonic Stem Cell Reporter Line Enables Identification of Human Basal Forebrain Derivatives

  2011cited by this paper
• Excitation-induced ataxin-3 aggregation in neurons from patients with Machado–Joseph disease

  2011cited by this paper
• Conversion of mouse and human fibroblasts into functional spinal motor neurons.

  2011cited by this paper
• Direct conversion of fibroblasts to functional neurons by defined factors

  2010cited by this paper
• Differential modeling of fragile X syndrome by human embryonic stem cells and induced pluripotent stem cells.

  2010cited by this paper
• Generation of neural crest cells and peripheral sensory neurons from human embryonic stem cells.

  2010cited by this paper
• Derivation of neural crest cells from human pluripotent stem cells

  2010cited by this paper
• Prospective Isolation of Cortical Interneuron Precursors from Mouse Embryonic Stem Cells

  2010cited by this paper
• The neurobiology of amyotrophic lateral sclerosis

  2010cited by this paper
• Murine Embryonic Stem Cell-Derived Pyramidal Neurons Integrate into the Cerebral Cortex and Appropriately Project Axons to Subcortical Targets

  2010cited by this paper
• Efficient generation of mature cerebellar Purkinje cells from mouse embryonic stem cells

  2010cited by this paper
• Differentiated Parkinson patient-derived induced pluripotent stem cells grow in the adult rodent brain and reduce motor asymmetry in Parkinsonian rats

  2010cited by this paper
• Olfactory Stem Cells, a New Cellular Model for Studying Molecular Mechanisms Underlying Familial Dysautonomia

  2010cited by this paper
• β-Amyloid 1-42 Oligomers Impair Function of Human Embryonic Stem Cell-Derived Forebrain Cholinergic Neurons

  2010cited by this paper
• Generation of induced pluripotent stem cells from human terminally differentiated circulating T cells.

  2010cited by this paper
• Generation of Human Induced Pluripotent Stem Cells from Umbilical Cord Matrix and Amniotic Membrane Mesenchymal Cells*

  2010cited by this paper
• Huntington’s disease: pathogenesis to animal models

  2010cited by this paper
• Friedreich's ataxia induced pluripotent stem cells model intergenerational GAA⋅TTC triplet repeat instability.

  2010influential reference
• Characterization of Human Huntington's Disease Cell Model from Induced Pluripotent Stem Cells

  2010cited by this paper
• Functional diversity of ESC-derived motor neuron subtypes revealed through intraspinal transplantation.

  2010cited by this paper
• Efficient derivation of functional floor plate tissue from human embryonic stem cells.

  2010cited by this paper
• Highly efficient reprogramming to pluripotency and directed differentiation of human cells with synthetic modified mRNA.

  2010cited by this paper
• Efficient Differentiation of Human Embryonic Stem Cells into Functional Cerebellar-Like Cells

  2010cited by this paper
• Modeling Pathogenesis and Treatment of Familial Dysautonomia using Patient Specific iPSCs

  2009cited by this paper
• Generation of rat and human induced pluripotent stem cells by combining genetic reprogramming and chemical inhibitors.

  2009cited by this paper
• Glucocerebrosidase mutations in clinical and pathologically proven Parkinson's disease.

  2009cited by this paper
• Stem cell-derived motor neurons: applications and challenges in amyotrophic lateral sclerosis.

  2009cited by this paper
• Friedreich ataxia: The clinical picture

  2009cited by this paper
• Excision of Reprogramming Transgenes Improves the Differentiation Potential of iPS Cells Generated with a Single Excisable Vector

  2009cited by this paper
• Multicellular models of Friedreich ataxia

  2009cited by this paper
• Coordination of sonic hedgehog and Wnt signaling determines ventral and dorsal telencephalic neuron types from human embryonic stem cells

  2009cited by this paper
• Parkinson's disease patient-derived induced pluripotent stem cells free of viral reprogramming factors.

  2009cited by this paper
• Generation of cortical neurons from mouse embryonic stem cells

  2009cited by this paper
• piggyBac transposition reprograms fibroblasts to induced pluripotent stem cells

  2009cited by this paper
• Diagnosis and treatment of Friedreich ataxia: a European perspective

  2009cited by this paper
• Stoichiometric and temporal requirements of Oct4, Sox2, Klf4, and c-Myc expression for efficient human iPSC induction and differentiation

  2009cited by this paper
• A chemical platform for improved induction of human iPSCs

  2009cited by this paper
• Spinal muscular atrophy: why do low levels of survival motor neuron protein make motor neurons sick?

  2009cited by this paper
• Faculty Opinions recommendation of Human induced pluripotent stem cells free of vector and transgene sequences.

  2009cited by this paper
• Highly efficient neural conversion of human ES and iPS cells by dual inhibition of SMAD signaling

  2009cited by this paper
• Adenoviral Gene Delivery Can Reprogram Human Fibroblasts to Induced Pluripotent Stem Cells

  2009cited by this paper
• Induced pluripotent stem cells from a spinal muscular atrophy patient

  2009influential reference
• Parkinson's disease: from monogenic forms to genetic susceptibility factors.

  2009cited by this paper
• Generation of induced pluripotent stem cells from human cord blood using OCT4 and SOX2.

  2009cited by this paper
• Generation of induced pluripotent stem cells without Myc from mouse and human fibroblasts

  2008cited by this paper
• Disease-specific induced pluripotent stem cells.

  2008influential reference
• TDP-43 Mutations in Familial and Sporadic Amyotrophic Lateral Sclerosis

  2008cited by this paper
• New insights into the pathoanatomy of spinocerebellar ataxia type 3 (Machado–Joseph disease)

  2008cited by this paper
• MicroRNA regulation of cell lineages in mouse and human embryonic stem cells.

  2008cited by this paper
• Promotion of Reprogramming to Ground State Pluripotency by Signal Inhibition

  2008cited by this paper
• Self-organized formation of polarized cortical tissues from ESCs and its active manipulation by extrinsic signals.

  2008cited by this paper
• An intrinsic mechanism of corticogenesis from embryonic stem cells

  2008cited by this paper
• Animal models of Alzheimer's disease and frontotemporal dementia

  2008cited by this paper
• Early Development of the Central and Peripheral Nervous Systems Is Coordinated by Wnt and BMP Signals

  2008cited by this paper
• Mechanisms of neurodegeneration in Huntington’s disease

  2008cited by this paper
• Retroviral vector silencing during iPS cell induction: An epigenetic beacon that signals distinct pluripotent states

  2008cited by this paper
• A drug-inducible system for direct reprogramming of human somatic cells to pluripotency.

  2008cited by this paper
• Generation of Mouse Induced Pluripotent Stem Cells Without Viral Vectors

  2008cited by this paper

• Increased Neuronal Differentiation Efficiency in High Cell Density-Derived Induced Pluripotent Stem Cells

  2019cites this paper
• Future trends and possibilities of using induced pluripotent stem cells (iPSC) inregenerative medicine

  2017cites this paper
• iPSC-derived neurons as a higher-throughput readout for autism: promises and pitfalls.

  2014cites this paper