Modeling human disease with pluripotent stem cells: from genome association to function.
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Summary
The opportunities and challenges of combining gene-editing technologies, induced pluripotent stem cells, and genome-wide association as well as DNA sequencing studies are discussed and a workflow for interrogating the contribution of disease-associated candidate genetic variants to disease-relevant phenotypes is provided.
- Type
- article
- Published
- 2013-06-06
- Cited by
- 187
- References
- 94
- Access
- Open access
- OpenAlex
- https://openalex.org/W23746975
- Semantic Scholar
- https://api.semanticscholar.org/CorpusID:11447665
Keywords
Gravitational singularity, Geometry, Complex geometry, Mathematics, Analytic geometry
References
- Sleep Disorders and Sleep Deprivation
- Orexin receptors as therapeutic drug targets.
- Induced Pluripotent Stem Cells from Patients with Huntington’s Disease Show CAG Repeat Expansion Associated Phenotypes
- Searching for genetic determinants in the new millennium
- A knockout mouse resource for the biomedical research community
- Directed Differentiation of Pluripotent Stem Cells into Kidney
- Direct Lineage Conversions: Unnatural but useful?
- Distinct metabolic flow enables large-scale purification of mouse and human pluripotent stem cell-derived cardiomyocytes.
- Trisomy Correction in Down Syndrome Induced Pluripotent Stem Cells
- Impaired Intrinsic Immunity to HSV-1 in Human iPSC-Derived TLR3-Deficient CNS Cells
- Breaking the Code of DNA Binding Specificity of TAL-Type III Effectors
- Finding the missing heritability of complex diseases
- Stem cells and drug discovery: the beginning of a new era?
- A functionally characterized test set of human induced pluripotent stem cells
- Non–cell autonomous effect of glia on motor neurons in an embryonic stem cell–based ALS model
- Alzheimer Mechanisms and Therapeutic Strategies
- LRRK2 Mutant iPSC-Derived DA Neurons Demonstrate Increased Susceptibility to Oxidative Stress
- RNA-Guided Human Genome Engineering via Cas9
- Initial impact of the sequencing of the human genome
- Direct conversion of mouse fibroblasts to hepatocyte-like cells by defined factors
Cited by
- Databases and Collaboration Require Standards for Human Stem Cell Research
- Rescue of DNA-PK Signaling and T-Cell Differentiation by Targeted Genome Editing in a prkdc Deficient iPSC Disease Model
- Derivation of Diverse Hormone-Releasing Pituitary Cells from Human Pluripotent Stem Cells
- Stem Cells in Drug Development
- Patient-Specific Induced Pluripotent Stem Cells for Disease Modeling and Phenotypic Drug Discovery.
- Generation of CD44 gene-deficient mouse derived induced pluripotent stem cells
- Genetics of celiac disease.
- Use of cartilage derived from murine induced pluripotent stem cells for osteoarthritis drug screening
- Cerebellar Development and Autism Spectrum Disorder in Tuberous Sclerosis Complex
- Interaction of Salmonella enterica Serovar Typhimurium with Intestinal Organoids Derived from Human Induced Pluripotent Stem Cells
- Pluripotent Stem Cells and Skeletal Regeneration – Promise and Potential
- Reprogramming cancer cells: a novel approach for cancer therapy or a tool for disease-modeling?
- Human Pluripotent Stem Cell-Derived Retinal Ganglion Cells: Applications for the Study and Treatment of Optic Neuropathies
- New opportunities: harnessing induced pluripotency for discovery in diabetes and metabolism
- Small Molecule Screening in Human Induced Pluripotent Stem Cell-derived Terminal Cell Types*
- Path from schizophrenia genomics to biology: gene regulation and perturbation in neurons derived from induced pluripotent stem cells and genome editing
- Combinatorial analysis of developmental cues efficiently converts human pluripotent stem cells into multiple neuronal subtypes
- iPSC crowdsourcing: a model for obtaining large panels of stem cell lines for screening.
- Pluripotent stem cell-based models to investigate retinal pigmented epithelium function and disease
- Fast clonal expansion and limited neural stem cell self-renewal in the adult subependymal zone
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