iPSC-Based Compound Screening and In Vitro Trials Identify a Synergistic Anti-amyloid β Combination for Alzheimer's Disease.
Sign Up to like & getrecommendations! Published in 2017 at "Cell reports"
DOI: 10.1016/j.celrep.2017.10.109
Abstract: In the process of drug development, in vitro studies do not always adequately predict human-specific drug responsiveness in clinical trials. Here, we applied the advantage of human iPSC-derived neurons, which offer human-specific drug responsiveness, to screen… read more here.
Keywords: drug; alzheimer disease; compound; ipsc based ... See more keywords
Bridging the Gaps: iPSC-Based Models from CHIP to MDS to AML.
Sign Up to like & getrecommendations! Published in 2017 at "Cell stem cell"
DOI: 10.1016/j.stem.2017.02.011
Abstract: Myeloid malignancies exist on a spectrum from asymptomatic clonal hematopoiesis to overt leukemia and exhibit substantial clonal heterogeneity. Both aspects are challenging to capture in experimental models. In two landmark studies in this issue of… read more here.
Keywords: gaps ipsc; based models; models chip; bridging gaps ... See more keywords
Development of iPSC-based clinical trial selection platform for patients with ultrarare diseases
Sign Up to like & getrecommendations! Published in 2022 at "Science Advances"
DOI: 10.1126/sciadv.abl4370
Abstract: A “Leap-of-Faith” approach is used to treat patients with previously unknown ultrarare pathogenic mutations, often based on evidence from patients having dissimilar but more prevalent mutations. This uncertainty reflects the need to develop personalized prescreening… read more here.
Keywords: platform; patient; development ipsc; clinical trial ... See more keywords
Elucidating an Essential Role of Promoting Endothelial-to-Hematopoietic Transition By a Key EMT-Regulator ZEB2 Using a Human iPSC-Based Model
Sign Up to like & getrecommendations! Published in 2019 at "Blood"
DOI: 10.1182/blood-2019-126908
Abstract: ZEB2 (Zinc finger E-box binding homeobox 2; also known as SMAD interacting protein-1 or SIP1) is a DNA-binding transcriptional regulator associated with epithelial-to-mesenchymal-transition (EMT). It has been reported that it is important in many other… read more here.
Keywords: zeb2; formation; model; ipsc ... See more keywords
Copy number variants (CNVs): a powerful tool for iPSC-based modelling of ASD
Sign Up to like & getrecommendations! Published in 2020 at "Molecular Autism"
DOI: 10.1186/s13229-020-00343-4
Abstract: Patients diagnosed with chromosome microdeletions or duplications, known as copy number variants (CNVs), present a unique opportunity to investigate the relationship between patient genotype and cell phenotype. CNVs have high genetic penetrance and give a… read more here.
Keywords: number variants; copy number; variants cnvs; ipsc based ... See more keywords
iPSC‐based modeling of THD recapitulates disease phenotypes and reveals neuronal malformation
Sign Up to like & getrecommendations! Published in 2022 at "EMBO Molecular Medicine"
DOI: 10.15252/emmm.202215847
Abstract: Tyrosine hydroxylase deficiency (THD) is a rare genetic disorder leading to dopaminergic depletion and early-onset parkinsonism. Affected children present with either a severe form that does not respond to L-Dopa treatment (THD-B), or a milder… read more here.
Keywords: thd; ipsc dan; dan; ipsc based ... See more keywords
Human-Origin iPSC-Based Recellularization of Decellularized Whole Rat Livers
Sign Up to like & getrecommendations! Published in 2022 at "Bioengineering"
DOI: 10.3390/bioengineering9050219
Abstract: End-stage liver diseases lead to mortality of millions of patients, as the only treatment available is liver transplantation and donor scarcity means that patients have to wait long periods before receiving a new liver. In… read more here.
Keywords: liver; rat; differentiation; ipsc based ... See more keywords
Progress in iPSC-Based Modeling of Psychiatric Disorders
Sign Up to like & getrecommendations! Published in 2019 at "International Journal of Molecular Sciences"
DOI: 10.3390/ijms20194896
Abstract: Progress in iPSC-based cellular systems provides new insights into human brain development and early neurodevelopmental deviations in psychiatric disorders. Among these, studies on schizophrenia (SCZ) take a prominent role owing to its high heritability and… read more here.
Keywords: psychiatric disorders; based modeling; progress ipsc; development ... See more keywords
Human iPSC-Based Modeling of Central Nerve System Disorders for Drug Discovery
Sign Up to like & getrecommendations! Published in 2021 at "International Journal of Molecular Sciences"
DOI: 10.3390/ijms22031203
Abstract: A high-throughput drug screen identifies potentially promising therapeutics for clinical trials. However, limitations that persist in current disease modeling with limited physiological relevancy of human patients skew drug responses, hamper translation of clinical efficacy, and… read more here.
Keywords: central nerve; drug; nerve system; drug discovery ... See more keywords