Concise Review: Patient-Derived Stem Cell Research for Monogenic Disorders (Q38557791)

Height matters-from monogenic disorders to normal variation

21 January 2018

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Gene therapy as a new treatment option for inherited monogenic diseases

21 January 2018

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Preimplantation Genetic Screening: An Effective Testing for Infertile and Repeated Miscarriage Patients?

21 January 2018

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Evaluation of targeted next-generation sequencing-based preimplantation genetic diagnosis of monogenic disease

21 January 2018

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The experience of 3 years of external quality assessment of preimplantation genetic diagnosis for cystic fibrosis

21 January 2018

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Blastocyst trophectoderm biopsy and preimplantation genetic diagnosis for familial monogenic disorders and chromosomal translocations.

21 January 2018

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Evaluation of PCR-based preimplantation genetic diagnosis applied to monogenic diseases: a collaborative ESHRE PGD consortium study.

21 January 2018

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Live birth after PGD with confirmation by a comprehensive approach (karyomapping) for simultaneous detection of monogenic and chromosomal disorders

21 January 2018

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Genetic modulation of senescent phenotypes in Homo sapiens

21 January 2018

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Developmental potential and dynamic behavior of hematopoietic stem cells.

21 January 2018

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In vivo clonal analysis reveals self-renewing and multipotent adult neural stem cell characteristics

21 January 2018

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Transcription factor achaete scute-like 2 controls intestinal stem cell fate

21 January 2018

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A gutsy way to grow: intestinal stem cells as nutrient sensors.

21 January 2018

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The intestinal crypt, a prototype stem cell compartment

21 January 2018

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A subpopulation of adult skeletal muscle stem cells retains all template DNA strands after cell division

21 January 2018

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Adult c-kit(pos) cardiac stem cells are necessary and sufficient for functional cardiac regeneration and repair.

21 January 2018

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Isolation of a pluripotent cell line from early mouse embryos cultured in medium conditioned by teratocarcinoma stem cells

21 January 2018

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Embryonic stem cell lines derived from human blastocysts

21 January 2018

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Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors

21 January 2018

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

21 January 2018

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Control of the embryonic stem cell state

21 January 2018

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The promise of induced pluripotent stem cells in research and therapy

21 January 2018

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PGD-derived human embryonic stem cell lines as a powerful tool for the study of human genetic disorders.

21 January 2018

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Epigenetic memory in induced pluripotent stem cells

21 January 2018

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Different developmental potential of pluripotent stem cells generated by different reprogramming strategies.

21 January 2018

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Abnormalities in human pluripotent cells due to reprogramming mechanisms

21 January 2018

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Enhanced telomere rejuvenation in pluripotent cells reprogrammed via nuclear transfer relative to induced pluripotent stem cells

21 January 2018

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Human embryonic stem cells derived by somatic cell nuclear transfer.

21 January 2018

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Human somatic cell nuclear transfer using adult cells.

21 January 2018

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Human oocytes reprogram adult somatic nuclei of a type 1 diabetic to diploid pluripotent stem cells

21 January 2018

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Induced pluripotent stem cell lines derived from human somatic cells

21 January 2018

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Induced pluripotent stem cells generated without viral integration

21 January 2018

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Adenoviral gene delivery can reprogram human fibroblasts to induced pluripotent stem cells

21 January 2018

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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

21 January 2018

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Generation of human-induced pluripotent stem cells by a nonintegrating RNA Sendai virus vector in feeder-free or xeno-free conditions

21 January 2018

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Generation of mouse induced pluripotent stem cells without viral vectors

21 January 2018

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Efficient human iPS cell derivation by a non-integrating plasmid from blood cells with unique epigenetic and gene expression signatures

21 January 2018

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Highly efficient reprogramming to pluripotency and directed differentiation of human cells with synthetic modified mRNA.

21 January 2018

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A comparison of non-integrating reprogramming methods

21 January 2018

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Generation of induced pluripotent stem cells using recombinant proteins

21 January 2018

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Generation of human induced pluripotent stem cells by direct delivery of reprogramming proteins

21 January 2018

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Pluripotent stem cells induced from mouse somatic cells by small-molecule compounds

21 January 2018

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Modeling the mitochondrial cardiomyopathy of Barth syndrome with induced pluripotent stem cell and heart-on-chip technologies

21 January 2018

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Induced pluripotent stem cell reprogramming by integration-free Sendai virus vectors from peripheral blood of patients with craniometaphyseal dysplasia

21 January 2018

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Recessive cardiac phenotypes in induced pluripotent stem cell models of Jervell and Lange-Nielsen syndrome: disease mechanisms and pharmacological rescue

21 January 2018

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Morphological features of iPS cells generated from Fabry disease skin fibroblasts using Sendai virus vector (SeVdp).

21 January 2018

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Wiskott-Aldrich syndrome iPS cells produce megakaryocytes with defects in cytoskeletal rearrangement and proplatelet formation.

21 January 2018

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Statin treatment rescues FGFR3 skeletal dysplasia phenotypes.

21 January 2018

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

21 January 2018

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Derivation, culture, and characterization of VUB hESC lines

21 January 2018

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Normal collagen and bone production by gene-targeted human osteogenesis imperfecta iPSCs

21 January 2018

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Exogenous activation of BMP-2 signaling overcomes TGFβ-mediated inhibition of osteogenesis in Marfan embryonic stem cells and Marfan patient-specific induced pluripotent stem cells.

21 January 2018

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Human embryonic stem cell lines with genetic disorders

21 January 2018

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Repository of human embryonic stem cell lines and development of individual specific lines using stembrid technology

21 January 2018

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Skeletogenic phenotype of human Marfan embryonic stem cells faithfully phenocopied by patient-specific induced-pluripotent stem cells

21 January 2018

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Derivation of human embryonic stem cell lines from embryos obtained after IVF and after PGD for monogenic disorders.

21 January 2018

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Laser-assisted derivation of human embryonic stem cell lines from IVF embryos after preimplantation genetic diagnosis

21 January 2018

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Length-dependent CTG·CAG triplet-repeat expansion in myotonic dystrophy patient-derived induced pluripotent stem cells.

21 January 2018

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Human embryonic stem cell models of Huntington disease.

21 January 2018

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High-efficiency derivation of human embryonic stem cell lines following pre-implantation genetic diagnosis.

21 January 2018

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Neuronal properties, in vivo effects, and pathology of a Huntington's disease patient-derived induced pluripotent stem cells

21 January 2018

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Inhibition of soluble tumor necrosis factor is therapeutic in Huntington's disease

21 January 2018

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Disease-specific induced pluripotent stem cells

21 January 2018

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Genetic correction of Huntington's disease phenotypes in induced pluripotent stem cells

21 January 2018

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Induced pluripotent stem cells from patients with Huntington's disease show CAG-repeat-expansion-associated phenotypes

21 January 2018

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Inhibition of mitochondrial fragmentation diminishes Huntington's disease-associated neurodegeneration

21 January 2018

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Successful disease-specific induced pluripotent stem cell generation from patients with kidney transplantation

21 January 2018

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Reduced ciliary polycystin-2 in induced pluripotent stem cells from polycystic kidney disease patients with PKD1 mutations

21 January 2018

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Directed differentiation of human pluripotent cells to ureteric bud kidney progenitor-like cells

21 January 2018

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

21 January 2018

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

21 January 2018

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Disease-corrected hepatocyte-like cells from familial hypercholesterolemia-induced pluripotent stem cells

21 January 2018

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Patient-specific induced pluripotent stem-cell models for long-QT syndrome

21 January 2018

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Using iPSC-derived neurons to uncover cellular phenotypes associated with Timothy syndrome

21 January 2018

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Alteration in basal and depolarization induced transcriptional network in iPSC derived neurons from Timothy syndrome.

21 January 2018

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

21 January 2018

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Seamless gene correction of β-thalassemia mutations in patient-specific iPSCs using CRISPR/Cas9 and piggyBac

21 January 2018

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Improved hematopoietic differentiation efficiency of gene-corrected beta-thalassemia induced pluripotent stem cells by CRISPR/Cas9 system

21 January 2018

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Induced pluripotent stem cells offer new approach to therapy in thalassemia and sickle cell anemia and option in prenatal diagnosis in genetic diseases

21 January 2018

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

21 January 2018

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Generation of human β-thalassemia induced pluripotent stem cells from amniotic fluid cells using a single excisable lentiviral stem cell cassette

21 January 2018

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Parallel assessment of globin lentiviral transfer in induced pluripotent stem cells and adult hematopoietic stem cells derived from the same transplanted β-thalassemia patient

21 January 2018

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Transcription activator-like effector nuclease (TALEN)-mediated gene correction in integration-free β-thalassemia induced pluripotent stem cells

21 January 2018

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Generation of human β-thalassemia induced pluripotent cell lines by reprogramming of bone marrow-derived mesenchymal stromal cells using modified mRNA.

21 January 2018

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Erythropoietic differentiation of a human embryonic stem cell line harbouring the sickle cell anaemia mutation

21 January 2018

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

21 January 2018

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Targeted gene correction minimally impacts whole-genome mutational load in human-disease-specific induced pluripotent stem cell clones.

21 January 2018

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In situ genetic correction of the sickle cell anemia mutation in human induced pluripotent stem cells using engineered zinc finger nucleases

21 January 2018

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Induced pluripotent stem cells from a spinal muscular atrophy patient

21 January 2018

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Brief report: phenotypic rescue of induced pluripotent stem cell-derived motoneurons of a spinal muscular atrophy patient.

21 January 2018

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Generation of induced pluripotent stem cells from Asian patients with chronic neurodegenerative diseases

21 January 2018

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Genetic correction of human induced pluripotent stem cells from patients with spinal muscular atrophy

21 January 2018

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Generation of a human embryonic stem cell line encoding the cystic fibrosis mutation deltaF508, using preimplantation genetic diagnosis

21 January 2018

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Derivation and characterization of three new Spanish human embryonic stem cell lines (VAL -3 -4 -5) on human feeder and in serum-free conditions.

21 January 2018

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Directed differentiation of human pluripotent stem cells into mature airway epithelia expressing functional CFTR protein

21 January 2018

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TALEN-mediated generation and genetic correction of disease-specific human induced pluripotent stem cells

21 January 2018

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Generation of transgene-free lung disease-specific human induced pluripotent stem cells using a single excisable lentiviral stem cell cassette.

21 January 2018

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Hematopoietic differentiation of induced pluripotent stem cells from patients with mucopolysaccharidosis type I (Hurler syndrome)

21 January 2018

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

21 January 2018

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Generation of liver disease-specific induced pluripotent stem cells along with efficient differentiation to functional hepatocyte-like cells

21 January 2018

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Establishment and directed differentiation of induced pluripotent stem cells from glycogen storage disease type Ib patient

21 January 2018

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

21 January 2018

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Pompe disease results in a Golgi-based glycosylation deficit in human induced pluripotent stem cell-derived cardiomyocytes.

21 January 2018

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The generation of induced pluripotent stem cells (iPSCs) from patients with infantile and late-onset types of Pompe disease and the effects of treatment with acid-α-glucosidase in Pompe's iPSCs

21 January 2018

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A human iPSC model of Hutchinson Gilford Progeria reveals vascular smooth muscle and mesenchymal stem cell defects

21 January 2018

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

21 January 2018

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Generation of induced pluripotent stem cell lines from 3 distinct laminopathies bearing heterogeneous mutations in lamin A/C

21 January 2018

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An upregulation in the expression of vanilloid transient potential channels 2 enhances hypotonicity-induced cytosolic Ca²⁺ rise in human induced pluripotent stem cell model of Hutchinson-Gillford Progeria.

21 January 2018

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Induced pluripotent stem cells reveal functional differences between drugs currently investigated in patients with hutchinson-gilford progeria syndrome

21 January 2018

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CEP290 gene transfer rescues Leber congenital amaurosis cellular phenotype.

21 January 2018

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Precise correction of the dystrophin gene in duchenne muscular dystrophy patient induced pluripotent stem cells by TALEN and CRISPR-Cas9.

21 January 2018

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Complete genetic correction of ips cells from Duchenne muscular dystrophy

21 January 2018

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Disease-specific induced pluripotent stem cells: a platform for human disease modeling and drug discovery

21 January 2018

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Myogenic differentiation of muscular dystrophy-specific induced pluripotent stem cells for use in drug discovery

21 January 2018

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Exon skipping and gene transfer restore dystrophin expression in human induced pluripotent stem cells-cardiomyocytes harboring DMD mutations

21 January 2018

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Modeling induced pluripotent stem cells from fibroblasts of Duchenne muscular dystrophy patients

21 January 2018

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Dystrophin-deficient cardiomyocytes derived from human urine: new biologic reagents for drug discovery.

21 January 2018

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Derivation and functional analysis of patient-specific induced pluripotent stem cells as an in vitro model of chronic granulomatous disease

21 January 2018

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

21 January 2018

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An AAVS1-targeted minigene platform for correction of iPSCs from all five types of chronic granulomatous disease

21 January 2018

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Optimized Generation of Functional Neutrophils and Macrophages from Patient-Specific Induced Pluripotent Stem Cells: Ex Vivo Models of X(0)-Linked, AR22(0)- and AR47(0)- Chronic Granulomatous Diseases

21 January 2018

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Modeling of hemophilia A using patient-specific induced pluripotent stem cells derived from urine cells

21 January 2018

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Rescue of ATP7B function in hepatocyte-like cells from Wilson's disease induced pluripotent stem cells using gene therapy or the chaperone drug curcumin

21 January 2018

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

21 January 2018

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Developmental study of fragile X syndrome using human embryonic stem cells derived from preimplantation genetically diagnosed embryos

21 January 2018

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Differential modeling of fragile X syndrome by human embryonic stem cells and induced pluripotent stem cells

21 January 2018

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Epigenetic characterization of the FMR1 gene and aberrant neurodevelopment in human induced pluripotent stem cell models of fragile X syndrome

21 January 2018

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Molecular analysis of FMR1 reactivation in fragile-X induced pluripotent stem cells and their neuronal derivatives

21 January 2018

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Signaling defects in iPSC-derived fragile X premutation neurons

21 January 2018

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Molecular mechanisms regulating the defects in fragile X syndrome neurons derived from human pluripotent stem cells

21 January 2018

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

21 January 2018

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Isogenic pairs of wild type and mutant induced pluripotent stem cell (iPSC) lines from Rett syndrome patients as in vitro disease model

21 January 2018

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Isolation of MECP2-null Rett Syndrome patient hiPS cells and isogenic controls through X-chromosome inactivation

21 January 2018

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Neuronal maturation defect in induced pluripotent stem cells from patients with Rett syndrome

21 January 2018

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iPS cells to model CDKL5-related disorders.

21 January 2018

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GluD1 is a common altered player in neuronal differentiation from both MECP2-mutated and CDKL5-mutated iPS cells

21 January 2018

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Cardiolipin deficiency affects respiratory chain function and organization in an induced pluripotent stem cell model of Barth syndrome

21 January 2018

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Induced pluripotent stem cell models from X-linked adrenoleukodystrophy patients

21 January 2018

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The gene expression profiles of induced pluripotent stem cells from individuals with childhood cerebral adrenoleukodystrophy are consistent with proposed mechanisms of pathogenesis

21 January 2018

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Lymphoid regeneration from gene-corrected SCID-X1 subject-derived iPSCs.

21 January 2018

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

21 January 2018

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Human ES- and iPS-derived myogenic progenitors restore DYSTROPHIN and improve contractility upon transplantation in dystrophic mice.

21 January 2018

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Cardiac repair in a porcine model of acute myocardial infarction with human induced pluripotent stem cell-derived cardiovascular cells

21 January 2018

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Human and mouse adipose-derived cells support feeder-independent induction of pluripotent stem cells

21 January 2018

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Generation of embryonic stem cells from mouse adipose-tissue derived cells via somatic cell nuclear transfer

21 January 2018

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Presenilin-1 L166P mutant human pluripotent stem cell-derived neurons exhibit partial loss of γ-secretase activity in endogenous amyloid-β generation.

21 January 2018

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Developing high-fidelity hepatotoxicity models from pluripotent stem cells

21 January 2018

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Accurate prediction of drug-induced liver injury using stem cell-derived populations

21 January 2018

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Prediction of interindividual differences in hepatic functions and drug sensitivity by using human iPS-derived hepatocytes.

21 January 2018

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Gene targeting in stem cells from individuals with osteogenesis imperfecta

21 January 2018

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ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering

21 January 2018

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Unexpected failure rates for modular assembly of engineered zinc fingers

21 January 2018

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DNA-binding specificity is a major determinant of the activity and toxicity of zinc-finger nucleases

21 January 2018

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A guide to genome engineering with programmable nucleases

21 January 2018

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Seamless correction of the sickle cell disease mutation of the HBB gene in human induced pluripotent stem cells using TALENs

21 January 2018

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A TALE nuclease architecture for efficient genome editing

21 January 2018

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

21 January 2018

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Targeted gene disruption in somatic zebrafish cells using engineered TALENs

21 January 2018

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Targeted genome editing across species using ZFNs and TALENs

21 January 2018

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A transcription activator-like effector toolbox for genome engineering

21 January 2018

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One-step generation of mice carrying reporter and conditional alleles by CRISPR/Cas-mediated genome engineering

21 January 2018

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Simultaneous generation and germline transmission of multiple gene mutations in rat using CRISPR-Cas systems.

21 January 2018

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Generation of gene-modified cynomolgus monkey via Cas9/RNA-mediated gene targeting in one-cell embryos.

21 January 2018

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Enhanced efficiency of human pluripotent stem cell genome editing through replacing TALENs with CRISPRs

21 January 2018

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Functional repair of CFTR by CRISPR/Cas9 in intestinal stem cell organoids of cystic fibrosis patients

21 January 2018

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Analysis of off-target effects of CRISPR/Cas-derived RNA-guided endonucleases and nickases.

21 January 2018

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DNA targeting specificity of RNA-guided Cas9 nucleases

21 January 2018

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Targeted gene knockout by direct delivery of zinc-finger nuclease proteins

21 January 2018

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DNA nicks promote efficient and safe targeted gene correction

21 January 2018

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Efficient genome modification by CRISPR-Cas9 nickase with minimal off-target effects.

21 January 2018

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Improving CRISPR-Cas nuclease specificity using truncated guide RNAs

21 January 2018

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Restoration of human dystrophin following transplantation of exon-skipping-engineered DMD patient stem cells into dystrophic mice

21 January 2018

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Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy

21 January 2018

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A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency

21 January 2018

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Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector

21 January 2018

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Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

21 January 2018

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Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients

21 January 2018

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Modern management of chronic granulomatous disease

21 January 2018

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Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy

21 January 2018

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Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome.

21 January 2018

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Progress in gene therapy for primary immunodeficiencies using lentiviral vectors.

21 January 2018

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