Progress and prospects in stem cell therapy

One-Step Generation of Mice Carrying Mutations in Multiple Genes by CRISPR/Cas-Mediated Genome Engineering

(2013) Haoyi Wang et al.   CELL

RNA-Guided Human Genome Engineering via Cas9

(2013) P. Mali et al.   SCIENCE

Zinc-finger nuclease-mediated correction of  -thalassemia in iPS cells

(2012) C.-J. Chang et al.   BLOOD

Genetic correction of β-thalassemia patient-specific iPS cells and its use in improving hemoglobin production in irradiated SCID mice

(2012) Yixuan Wang et al.   CELL RESEARCH

Small Molecule-Mediated TGF-β Type II Receptor Degradation Promotes Cardiomyogenesis in Embryonic Stem Cells

(2012) Erik Willems et al.   Cell Stem Cell

Feasibility, Safety, and Therapeutic Efficacy of Human Induced Pluripotent Stem Cell-Derived Cardiomyocyte Sheets in a Porcine Ischemic Cardiomyopathy Model

(2012) M. Kawamura et al.   CIRCULATION

Extracellular Matrix Promotes Highly Efficient Cardiac Differentiation of Human Pluripotent Stem Cells

(2012) Jianhua Zhang et al.   CIRCULATION RESEARCH

Progressive degeneration of human neural stem cells caused by pathogenic LRRK2

(2012) Guang-Hui Liu et al.   NATURE

Heart repair by reprogramming non-myocytes with cardiac transcription factors

(2012) Kunhua Song et al.   NATURE

In vivo reprogramming of murine cardiac fibroblasts into induced cardiomyocytes

(2012) Li Qian et al.   NATURE

Donor-dependent variations in hepatic differentiation from human-induced pluripotent stem cells

(2012) M. Kajiwara et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Cozzarelli Prize Winner: Robust cardiomyocyte differentiation from human pluripotent stem cells via temporal modulation of canonical Wnt signaling

(2012) X. Lian et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

The Role of Allogeneic Hematopoietic Stem Cell Transplantation in the Therapy of Patients with Acute Lymphoblastic Leukemia

(2012) Partow Kebriaei et al.   Current Hematologic Malignancy Reports

Converted neural cells: induced to a cure?

(2012) Weiqi Zhang et al.   Protein & Cell

Establishment of hepatic and neural differentiation platforms of Wilson’s disease specific induced pluripotent stem cells

(2012) Fei Yi et al.   Protein & Cell

Oxidase-deficient neutrophils from X-linked chronic granulomatous disease iPS cells: functional correction by zinc finger nuclease-mediated safe harbor targeting

(2011) J. Zou 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

Generation of Isogenic Pluripotent Stem Cells Differing Exclusively at Two Early Onset Parkinson Point Mutations

(2011) Frank Soldner et al.   CELL

Efficient correction of hemoglobinopathy-causing mutations by homologous recombination in integration-free patient iPSCs

(2011) Mo Li et al.   CELL RESEARCH

Functional Integration of Dopaminergic Neurons Directly Converted from Mouse Fibroblasts

(2011) Jongpil Kim et al.   Cell Stem Cell

Targeted Gene Correction of Laminopathy-Associated LMNA Mutations in Patient-Specific iPSCs

(2011) Guang-Hui Liu et al.   Cell Stem Cell

Rapid generation of mature hepatocyte-like cells from human induced pluripotent stem cells by an efficient three-step protocol

(2011) Yu-Fan Chen et al.   HEPATOLOGY

Directed Evolution of Adeno-associated Virus for Enhanced Gene Delivery and Gene Targeting in Human Pluripotent Stem Cells

(2011) Prashanth Asuri et al.   MOLECULAR THERAPY

Recapitulation of premature ageing with iPSCs from Hutchinson–Gilford progeria syndrome

(2011) Guang-Hui Liu et al.   NATURE

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

(2011) Kosuke Yusa et al.   NATURE

Direct generation of functional dopaminergic neurons from mouse and human fibroblasts

(2011) Massimiliano Caiazzo et al.   NATURE

Genetic engineering of human pluripotent cells using TALE nucleases

(2011) Dirk Hockemeyer et al.   NATURE BIOTECHNOLOGY

An unbiased genome-wide analysis of zinc-finger nuclease specificity

(2011) Richard Gabriel et al.   NATURE BIOTECHNOLOGY

Site-specific integration and tailoring of cassette design for sustainable gene transfer

(2011) Angelo Lombardo et al.   NATURE METHODS

Revealing off-target cleavage specificities of zinc-finger nucleases by in vitro selection

(2011) Vikram Pattanayak et al.   NATURE METHODS

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

(2011) Claudio Mussolino et al.   NUCLEIC ACIDS RESEARCH

Genetic correction and analysis of induced pluripotent stem cells from a patient with gyrate atrophy

(2011) S. E. Howden et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

Potential Clinical Applications for Human Pluripotent Stem Cell-Derived Blood Components

(2011) Erin A. Kimbrel et al.   Stem Cells International

Functional genomics, proteomics, and regulatory DNA analysis in isogenic settings using zinc finger nuclease-driven transgenesis into a safe harbor locus in the human genome

(2010) R. C. DeKelver et al.   GENOME RESEARCH

Genomic safe harbors permit high β-globin transgene expression in thalassemia induced pluripotent stem cells

(2010) Eirini P Papapetrou et al.   NATURE BIOTECHNOLOGY

A TALE nuclease architecture for efficient genome editing

(2010) Jeffrey C Miller et al.   NATURE BIOTECHNOLOGY

Differentiation and Characterization of Metabolically Functioning Hepatocytes from Human Embryonic Stem Cells

(2010) Yuyou Duan et al.   STEM CELLS

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

(2009) Jizhong Zou et al.   Cell Stem Cell

Non–cell autonomous toxicity in neurodegenerative disorders: ALS and beyond

(2009) Hristelina Ilieva et al.   JOURNAL OF CELL BIOLOGY

Disease-corrected haematopoietic progenitors from Fanconi anaemia induced pluripotent stem cells

(2009) Ángel Raya 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

Human cardiovascular progenitor cells develop from a KDR+ embryonic-stem-cell-derived population

(2008) Lei Yang et al.   NATURE

Dopamine neurons implanted into people with Parkinson's disease survive without pathology for 14 years

(2008) Ivar Mendez et al.   NATURE MEDICINE

Highly efficient transient gene expression and gene targeting in primate embryonic stem cells with helper-dependent adenoviral vectors

(2008) K. Suzuki et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Neurons derived from reprogrammed fibroblasts functionally integrate into the fetal brain and improve symptoms of rats with Parkinson's disease

(2008) M. Wernig et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Efficient Differentiation of Functional Hepatocytes from Human Embryonic Stem Cells

(2008) Sadhana Agarwal et al.   STEM CELLS