At ASCB 2015: CRISPR/Cas9 + HPSC = human PKD lab model

(American Society for Cell Biology) By linking up CRISPR/Cas9 with another cutting edge technology, human pluripotent stem cells (hPSCs), Benjamin Freedman, now at the University of Washington, and his colleagues in Joseph Bonventre's lab at Harvard Medical School, have used CRISPR/Cas9 to guide hPSCs into becoming a human cell-based lab model system for polycystic kidney disease (PKD).
Source: EurekAlert! - Medicine and Health - Category: Global & Universal Source Type: news

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Publication date: Available online 10 August 2018Source: Stem Cell ResearchAuthor(s): Caroline Amalie Brunbjerg Hey, Katarina Beata Saltõkowa, Lasse Jonsgaard Larsen, Zeynep Tümer, Karen Brøndum-Nielsen, Karen Grønskov, Tina Duelund Hjortshøj, Lisbeth Birk MøllerAbstractBardet-Biedl syndrome (BBS) is an autosomal recessive ciliopathy with a wide range of symptoms including obesity, retinal dystrophy, polycystic kidney disease, polydactyly, hypogonadism and learning difficulties. Here we describe the successful generation of an induced pluripotent stem cell (iPSC) KCi001-A from a BBS ...
Source: Stem Cell Research - Category: Stem Cells Source Type: research
Publication date: June 2018Source: American Journal of Kidney Diseases, Volume 71, Issue 6Author(s): Nelly M. Cruz, Benjamin S. FreedmanCRISPR is a nuclease guidance system that enables rapid and efficient gene editing of specific DNA sequences within genomes. We review applications of CRISPR for the study and treatment of kidney disease. CRISPR enables functional experiments in cell lines and model organisms to validate candidate genes arising from genetic studies. CRISPR has furthermore been used to establish the first models of genetic disease in human kidney organoids derived from pluripotent stem cells. These gene-edi...
Source: American Journal of Kidney Diseases - Category: Urology & Nephrology Source Type: research
Abstract Krüppel-like zinc finger proteins form one of the largest families of transcription factors. They function as key regulators of embryonic development and a wide range of other physiological processes, and are implicated in a variety of pathologies. GLI-similar 1-3 (GLIS1-3) constitute a subfamily of Krüppel-like zinc finger proteins that act either as activators or repressors of gene transcription. GLIS3 plays a critical role in the regulation of multiple biological processes and is a key regulator of pancreatic β cell generation and maturation, insulin gene expression, thyroid hormone bios...
Source: Cellular and Molecular Life Sciences : CMLS - Category: Cytology Authors: Tags: Cell Mol Life Sci Source Type: research
Abstract Organoids derived from human pluripotent stem cells are a potentially powerful tool for high-throughput screening (HTS), but the complexity of organoid cultures poses a significant challenge for miniaturization and automation. Here, we present a fully automated, HTS-compatible platform for enhanced differentiation and phenotyping of human kidney organoids. The entire 21-day protocol, from plating to differentiation to analysis, can be performed automatically by liquid-handling robots, or alternatively by manual pipetting. High-content imaging analysis reveals both dose-dependent and threshold effects duri...
Source: Cell Stem Cell - Category: Stem Cells Authors: Tags: Cell Stem Cell Source Type: research
In January, 2018, Academic Press published my bookPrecision Medicine and the Reinvention of Human Disease. This book has an excellent " look inside " at itsGoogle book site, which includes the Table of Contents. In addition, I thought it might be helpful to see the topics listed in the Book's index. Note that page numbers followed by f indicate figures, t indicate tables, and ge indicate glossary terms.AAbandonware, 270, 310geAb initio, 34, 48ge, 108geABL (abelson leukemia) gene, 28, 58ge, 95 –97Absidia corymbifera, 218Acanthameoba, 213Acanthosis nigricans, 144geAchondroplasia, 74, 143ge, 354geAcne, 54ge, 1...
Source: Specified Life - Category: Information Technology Tags: index jules berman jules j berman precision medicine Source Type: blogs
Abstract Autophagy is a process essential for cell survival under stress condition. The patients with autosomal dominant polycystic kidney disease, which is caused by polycystin‐1 or polycystin‐2 (PKD2) mutation, display cardiovascular abnormalities and dysregulation in autophagy. However, it is unclear whether PKD2 plays a role in autophagy. In the present study, we explored the functional role of PKD2 in autophagy and apoptosis in human embryonic stem cell‐derived cardiomyocytes. HES2 hESC line‐derived cardiomyocytes (HES2‐CMs) were transduced with adenoviral‐based PKD2‐shRNAs (Ad‐PKD2‐shRNAs), and then...
Source: Stem Cells - Category: Stem Cells Authors: Tags: Embryonic Stem Cells/Induced Pluripotent Stem Cells Source Type: research
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Source: Stem Cells - Category: Stem Cells Authors: Tags: Embryonic Stem Cells/Induced Pluripotent Stem Cells Source Type: research
CONCLUSION: A number of pharmacotherapy approaches to ADPKD management show promise but are unlikely to be curative, fueling interest among researchers in finding new applications for nanomedicine and stem cell technologies that can slow ADPKD progression and better control complications of the disease. PMID: 29167138 [PubMed - in process]
Source: American Journal of Health-System Pharmacy : AJHP - Category: Drugs & Pharmacology Authors: Tags: Am J Health Syst Pharm Source Type: research
Publication date: December 2017 Source:Stem Cell Research, Volume 25 Author(s): Ming-Ching Ho, Ching-Ying Huang, Jia-Jung Lee, Shih-Han Hsu, Yu-Che Cheng, Yu-Hung Hsu, Daw-Yang Hwang, Huai-En Lu, Hung-Chun Chen, Patrick C.H. Hsieh Autosomal dominant polycystic kidney disease (ADPKD) is one of the most commonly inherited forms of polycystic kidney disease, and is characterized by the growth of numerous cysts in both kidneys. Here we generated an induced pluripotent stem cell (iPSC) line from the peripheral blood mononuclear cells (PBMCs) of a 63-year-old female ADPKD patient carrying an R803X mutation in the PKD2 gene usin...
Source: Stem Cell Research - Category: Stem Cells Source Type: research
Publication date: December 2017 Source:Stem Cell Research, Volume 25 Author(s): Jia-Jung Lee, Ming-Ching Ho, Ching-Ying Huang, Cheng-Hao Wen, Yu-Che Cheng, Yu-Hung Hsu, Daw-Yang Hwang, Huai-En Lu, Hung-Chun Chen, Patrick C.H. Hsieh Autosomal Dominant Polycystic Kidney Disease (ADPKD) is the most prevalent monogenic kidney disorder leading to kidney failure. We generated induced pluripotent stem cells (iPSCs) from a 37-year-old man carrying a PKD1 Q533X mutation who suffered from kidney failure and a myocardial infarction. The iPSCs were reprogrammed from the patient's peripheral blood mononuclear cells using the Sendai vi...
Source: Stem Cell Research - Category: Stem Cells Source Type: research
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