Efficient drug screening and gene correction for treating liver disease using patient-specific stem cells

Genetic and pharmacological disruption of the TEAD-YAP complex suppresses the oncogenic activity of YAP

(2012) Y. Liu-Chittenden et al.   GENES & DEVELOPMENT

JD induced pluripotent stem cell-derived hepatocytes faithfully recapitulate the pathophysiology of familial hypercholesterolemia

(2012) Max A. Cayo et al.   HEPATOLOGY

Applications of Patient-Specific Induced Pluripotent Stem Cells; Focused on Disease Modeling, Drug Screening and Therapeutic Potentials for Liver Disease

(2012) Yong Soon Chun et al.   International Journal of Biological Sciences

Histone Deacetylase Inhibitor (HDACi) Suberoylanilide Hydroxamic Acid (SAHA)-mediated Correction of α1-Antitrypsin Deficiency

(2012) Marion Bouchecareilh et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

In vivo genome editing using a high-efficiency TALEN system

(2012) Victoria M. Bedell et al.   NATURE

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

(2012) Lindsay Cade et al.   NUCLEIC ACIDS RESEARCH

Pluripotent Stem Cell-Based Cancer Therapy: Promise and Challenges

(2012) S. J. Sharkis et al.   Science Translational Medicine

Drug Screening for ALS Using Patient-Specific Induced Pluripotent Stem Cells

(2012) N. Egawa et al.   Science Translational Medicine

Efficient Derivation and Genetic Modifications of Human Pluripotent Stem Cells on Engineered Human Feeder Cell Lines

(2012) Chunlin Zou et al.   STEM CELLS AND DEVELOPMENT

Reprogramming of EBV-immortalized B-lymphocyte cell lines into induced pluripotent stem cells

(2011) S. M. Choi et al.   BLOOD

Site-specific gene correction of a point mutation in human iPS cells derived from an adult patient with sickle cell disease

(2011) J. Zou et al.   BLOOD

Liver engraftment potential of hepatic cells derived from patient-specific induced pluripotent stem cells

(2011) Su Mi Choi et al.   CELL CYCLE

Hematopoietic cells as sources for patient-specific iPSCs and disease modeling

(2011) Zhaohui Ye et al.   CELL CYCLE

Targeted gene correction of α1-antitrypsin deficiency in induced pluripotent stem cells

(2011) Kosuke Yusa et al.   NATURE

Genetic engineering of human pluripotent cells using TALE nucleases

(2011) Dirk Hockemeyer et al.   NATURE BIOTECHNOLOGY

Targeted gene disruption in somatic zebrafish cells using engineered TALENs

(2011) Jeffry D Sander et al.   NATURE BIOTECHNOLOGY

Efficient design and assembly of custom TALEN and other TAL effector-based constructs for DNA targeting

(2011) Tomas Cermak et al.   NUCLEIC ACIDS RESEARCH

Chemical screen identifies FDA-approved drugs and target pathways that induce precocious pancreatic endocrine differentiation

(2011) M. Rovira et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Targeted Genome Editing Across Species Using ZFNs and TALENs

(2011) Andrew J. Wood et al.   SCIENCE

In Vivo Liver Regeneration Potential of Human Induced Pluripotent Stem Cells from Diverse Origins

(2011) H. Liu et al.   Science Translational Medicine

In Situ Genetic Correction of the Sickle Cell Anemia Mutation in Human Induced Pluripotent Stem Cells Using Engineered Zinc Finger Nucleases

(2011) Vittorio Sebastiano et al.   STEM CELLS

A Novel Two-Stage, Transdisciplinary Study Identifies Digoxin as a Possible Drug for Prostate Cancer Treatment

(2011) Elizabeth A. Platz et al.   Cancer Discovery

Itraconazole, a Commonly Used Antifungal that Inhibits Hedgehog Pathway Activity and Cancer Growth

(2010) James Kim et al.   CANCER CELL

Generation of endoderm-derived human induced pluripotent stem cells from primary hepatocytes

(2010) Hua Liu et al.   HEPATOLOGY

Chemical Inducers of Autophagy That Enhance the Clearance of Mutant Proteins in Neurodegenerative Diseases

(2010) Maurizio Renna et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Modeling inherited metabolic disorders of the liver using human induced pluripotent stem cells

(2010) S. Tamir Rashid et al.   JOURNAL OF CLINICAL INVESTIGATION

Effect of Nitroxoline on Angiogenesis and Growth of Human Bladder Cancer

(2010) Joong Sup Shim et al.   JNCI-Journal of the National Cancer Institute

A TALE nuclease architecture for efficient genome editing

(2010) Jeffrey C Miller et al.   NATURE BIOTECHNOLOGY

Automated High-Content Live Animal Drug Screening Using C. elegans Expressing the Aggregation Prone Serpin α1-antitrypsin Z

(2010) Sager J. Gosai et al.   PLoS One

An Autophagy-Enhancing Drug Promotes Degradation of Mutant  1-Antitrypsin Z and Reduces Hepatic Fibrosis

(2010) T. Hidvegi et al.   SCIENCE

Human-induced pluripotent stem cells from blood cells of healthy donors and patients with acquired blood disorders

(2009) Z. Ye et al.   BLOOD

Gene Targeting of a Disease-Related Gene in Human Induced Pluripotent Stem and Embryonic Stem Cells

(2009) Jizhong Zou et al.   Cell Stem Cell

Recent Advances in Physicochemical and ADMET Profiling in Drug Discovery

(2009) Jianling Wang et al.   CHEMISTRY & BIODIVERSITY

HTS and hit finding in academia – from chemical genomics to drug discovery

(2009) Julie A. Frearson et al.   DRUG DISCOVERY TODAY

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

(2009) Gabsang Lee et al.   NATURE

Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases

(2009) Dirk Hockemeyer et al.   NATURE BIOTECHNOLOGY

Lessons from 60 years of pharmaceutical innovation

(2009) Bernard Munos   NATURE REVIEWS DRUG DISCOVERY

Autophagic disposal of the aggregation-prone protein that causes liver inflammation and carcinogenesis in α-1-antitrypsin deficiency

(2008) D H Perlmutter   CELL DEATH AND DIFFERENTIATION