Induced pluripotent stem cell technology for disease modeling and drug screening with emphasis on lysosomal storage diseases

A high-throughput FRET-based assay for determination of Atg4 activity

(2012) Min Li et al.   Autophagy

New windows to enhance direct reprogramming of somatic cells towards induced pluripotent stem cells

(2012) Saeideh Nakhaei-Rad et al.   Biochemistry and Cell Biology

The promise of induced pluripotent stem cells in research and therapy

(2012) Daisy A. Robinton et al.   NATURE

Chemical Strategies for Stem Cell Biology and Regenerative Medicine

(2011) Saiyong Zhu et al.   Annual Review of Biomedical Engineering

Identification and Characterization of Pharmacological Chaperones to Correct Enzyme Deficiencies in Lysosomal Storage Disorders

(2011) Kenneth J. Valenzano et al.   ASSAY AND DRUG DEVELOPMENT TECHNOLOGIES

Targeting SOX17 in Human Embryonic Stem Cells Creates Unique Strategies for Isolating and Analyzing Developing Endoderm

(2011) Pei Wang et al.   Cell Stem Cell

Reprogramming of Mouse and Human Cells to Pluripotency Using Mature MicroRNAs

(2011) Norikatsu Miyoshi et al.   Cell Stem Cell

Lacidipine Remodels Protein Folding and Ca2+ Homeostasis in Gaucher's Disease Fibroblasts: A Mechanism to Rescue Mutant Glucocerebrosidase

(2011) Fan Wang et al.   CHEMISTRY & BIOLOGY

Immunogenicity of Induced Pluripotent Stem Cells

(2011) Keisuke Okita et al.   CIRCULATION RESEARCH

Enzyme replacement therapy for lysosomal storage diseases

(2011) Robin H. Lachmann   CURRENT OPINION IN PEDIATRICS

Modeling neuronal defects associated with a lysosomal disorder using patient-derived induced pluripotent stem cells

(2011) Thomas Lemonnier et al.   HUMAN MOLECULAR GENETICS

Human Pompe disease-induced pluripotent stem cells for pathogenesis modeling, drug testing and disease marker identification

(2011) Hsiang-Po Huang et al.   HUMAN MOLECULAR GENETICS

Generation of induced pluripotent stem (iPS) cells derived from a murine model of Pompe disease and differentiation of Pompe-iPS cells into skeletal muscle cells

(2011) Shiho Kawagoe et al.   MOLECULAR GENETICS AND METABOLISM

Modelling the long QT syndrome with induced pluripotent stem cells

(2011) Ilanit Itzhaki et al.   NATURE

Molecular chaperones in protein folding and proteostasis

(2011) F. Ulrich Hartl et al.   NATURE

Direct conversion of human fibroblasts to dopaminergic neurons

(2011) U. Pfisterer et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Surface Marker Epithelial Cell Adhesion Molecule and E-cadherin Facilitate the Identification and Selection of Induced Pluripotent Stem Cells

(2011) Hsin-Fu Chen et al.   Stem Cell Reviews and Reports

Assessment of the Myogenic Stem Cell Compartment Following Transplantation ofPax3/Pax7-Induced Embryonic Stem Cell-Derived Progenitors

(2011) Radbod Darabi et al.   STEM CELLS

Suppression of autophagy permits successful enzyme replacement therapy in a lysosomal storage disorder—murine Pompe disease

(2010) Nina Raben et al.   Autophagy

A Model for Neural Development and Treatment of Rett Syndrome Using Human Induced Pluripotent Stem Cells

(2010) Maria C.N. Marchetto et al.   CELL

Differential Modeling of Fragile X Syndrome by Human Embryonic Stem Cells and Induced Pluripotent Stem Cells

(2010) Achia Urbach et al.   Cell Stem Cell

Friedreich's Ataxia Induced Pluripotent Stem Cells Model Intergenerational GAA⋅TTC Triplet Repeat Instability

(2010) Sherman Ku et al.   Cell Stem Cell

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

(2010) Luigi Warren et al.   Cell Stem Cell

Progress toward the clinical application of patient-specific pluripotent stem cells

(2010) Evangelos Kiskinis et al.   JOURNAL OF CLINICAL INVESTIGATION

Patient-specific induced pluripotent stem-cell-derived models of LEOPARD syndrome

(2010) Xonia Carvajal-Vergara et al.   NATURE

Telomere elongation in induced pluripotent stem cells from dyskeratosis congenita patients

(2010) Suneet Agarwal et al.   NATURE

Direct conversion of fibroblasts to functional neurons by defined factors

(2010) Thomas Vierbuchen et al.   NATURE

Human stem cells and drug screening: opportunities and challenges

(2010) Allison D. Ebert et al.   NATURE REVIEWS DRUG DISCOVERY

Cellular and in vivo activity of a novel PI3K inhibitor, PX-866, against human glioblastoma

(2010) D. Koul et al.   NEURO-ONCOLOGY

Patient-Specific Induced Pluripotent Stem-Cell Models for Long-QT Syndrome

(2010) Alessandra Moretti et al.   NEW ENGLAND JOURNAL OF MEDICINE

Lysosomal Storage Disease: Revealing Lysosomal Function and Physiology

(2010) Emma J. Parkinson-Lawrence et al.   PHYSIOLOGY

Induced pluripotent stem cells derived from mouse models of lysosomal storage disorders

(2010) Xing-Li Meng et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

High content screening: seeing is believing

(2010) Fabian Zanella et al.   TRENDS IN BIOTECHNOLOGY

Carnitine, mitochondrial function and therapy☆

(2009) Victor A. Zammit et al.   ADVANCED DRUG DELIVERY REVIEWS

Disease Models from Pluripotent Stem Cells

(2009) Claudia Lengerke et al.   Annals of the New York Academy of Sciences

Can cellular models revolutionize drug discovery in Parkinson's disease?

(2009) Birgitt Schüle et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE

Molecules that Promote or Enhance Reprogramming of Somatic Cells to Induced Pluripotent Stem Cells

(2009) Bo Feng et al.   Cell Stem Cell

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

(2009) Dohoon Kim et al.   Cell Stem Cell

Compounds from an unbiased chemical screen reverse both ER-to-Golgi trafficking defects and mitochondrial dysfunction in Parkinson's disease models

(2009) L. J. Su et al.   Disease Models & Mechanisms

Treating lysosomal storage diseases with pharmacological chaperones: from concept to clinics

(2009) Giancarlo Parenti   EMBO Molecular Medicine

The pharmacological chaperone 1-deoxynojirimycin increases the activity and lysosomal trafficking of multiple mutant forms of acid alpha-glucosidase

(2009) John J. Flanagan et al.   HUMAN MUTATION

Miglustat in patients with Niemann-Pick disease Type C (NP-C): A multicenter observational retrospective cohort study

(2009) M. Pineda et al.   MOLECULAR GENETICS AND METABOLISM

Modelling pathogenesis and treatment of familial dysautonomia using patient-specific iPSCs

(2009) Gabsang Lee et al.   NATURE

piggyBac transposition reprograms fibroblasts to induced pluripotent stem cells

(2009) Knut Woltjen et al.   NATURE

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

Chemical and Biological Approaches Synergize to Ameliorate Protein-Folding Diseases

(2008) Ting-Wei Mu et al.   CELL

Disease-Specific Induced Pluripotent Stem Cells

(2008) In-Hyun Park et al.   CELL

High-Throughput Screening Assay for the Identification of Compounds Regulating Self-Renewal and Differentiation in Human Embryonic Stem Cells

(2008) Sabrina C. Desbordes et al.   Cell Stem Cell

Lysosomal Storage Diseases and the Blood-Brain Barrier

(2008) David Begley et al.   CURRENT PHARMACEUTICAL DESIGN

Induced pluripotent stem cells from a spinal muscular atrophy patient

(2008) Allison D. Ebert et al.   NATURE

Induced Pluripotent Stem Cells Generated from Patients with ALS Can Be Differentiated into Motor Neurons

(2008) J. T. Dimos et al.   SCIENCE

Partial Restoration of Mutant Enzyme Homeostasis in Three Distinct Lysosomal Storage Disease Cell Lines by Altering Calcium Homeostasis

(2008) Ting-Wei Mu et al.   PLOS BIOLOGY

Efficient propagation of single cells accutase-dissociated human embryonic stem cells

(2007) Ruchi Bajpai et al.   MOLECULAR REPRODUCTION AND DEVELOPMENT