Patient-Specific Induced Pluripotent Stem Cell Models

Patient-Specific Induced Pluripotent Stem Cell Models
Author: Andras Nagy
Publisher: Humana
Total Pages: 0
Release: 2015-11-08
Genre: Science
ISBN: 9781493930333

This volume captures the rapid developments in the field of induced pluripotent stem (iPS) cells, which have provided novel opportunities and approaches both for better understanding a number of human diseases and for developing new platforms for drug development and screening for such diseases. Specifically, representative protocols on various disease models have been collected from labs around the world. Written for the highly successful Methods in Molecular Biology series, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and tips on troubleshooting and avoiding known pitfalls. Authoritative and practical, Patient-Specific Induced Pluripotent Stem Cell Models: Generation and Characterization is an ideal reference for scientists working on furthering iPS research.

Patient-Specific Stem Cells

Patient-Specific Stem Cells
Author: Deepak A. Lamba
Publisher: CRC Press
Total Pages: 279
Release: 2017-04-07
Genre: Medical
ISBN: 1315402173

One of the biggest challenges faced in medical research had been to create accurate and relevant models of human disease. A number of good animal models have been developed to understand the pathophysiology. However, not all of them reflect the human disorder, a classic case being Usher’s syndrome where the mutant mice do not have the same visual and auditory defects that patients face. There are others which have been even more difficult to model due to the multi-factorial nature of the condition and due to lack of discovery of a single causative gene such as age-related macular degeneration or Alzheimer’s syndrome. Thus a more relevant and accurate system will allow us to make better predictions on relevant therapeutic approaches. The discovery of human pluripotent stem cells in 1998 followed by the technological advances to reprogram somatic cells to pluripotent-stem cell-like cells in 2006 has completely revolutionized the way we can now think about modelling human development and disease. This now coupled with genome editing technologies such as TALENS and CRISPRs have now set us up to develop in vitro models both 2D as well as 3D organoids, which can more precisely reflect the disease in the patients. These combinatorial technologies are already providing us with better tools and therapeutics in drug discovery or gene therapy. This book summarizes both the technological advances in the field of generation of patient specific lines as well as various gene editing approaches followed by its applicability in various systems. The book will serve as a reference for the current state of the field as it: -Provides a comprehensive overview of the status of the field of patients derived induced pluripotent stem cells. -Describes the use of cardiac cells as a main featured component within the book. -Examines drug toxicity analysis as a working example throughout the book.

An Integrated Approach Using Patient-specific Induced Pluripotent Stem Cells and Protein Biochemistry to Study Vici Syndrome Associated Cardiomyopathy

An Integrated Approach Using Patient-specific Induced Pluripotent Stem Cells and Protein Biochemistry to Study Vici Syndrome Associated Cardiomyopathy
Author: Jing Qi
Publisher:
Total Pages:
Release: 2016
Genre:
ISBN:

Vici syndrome is a rare autosomal recessive inherited multisystem disorder caused by mutations in EPG5. More than 80% of the patients with Vici syndrome suffer from cardiomyopathy. In this study, the induced pluripotent stem cell (iPSC) system was applied to model the disease, investigate the cardiac phenotype and to elucidate the underlying molecular mechanism of autophagy in cardiomyocytes derived from iPSCs. HiPSCs from the patient with Vici syndrome (Vici-iPSCs) carrying a homologous intronic mutation of EPG5 (c.4952+1G>A) were generated. These Vici-iPSC lines were pluripotent and could...

Modeling Rett Syndrome with Patient-specific Induced Pluripotent Stem Cells

Modeling Rett Syndrome with Patient-specific Induced Pluripotent Stem Cells
Author:
Publisher:
Total Pages: 168
Release: 2013
Genre:
ISBN:

Rett syndrome (RTT) is one of the leading causes of intellectual disability in girls. Mutations in the X-linked methyl-CpG-binding protein 2 (MECP2) gene have been identified as the cause of RTT. Mouse and patient-derived induced pluripotent stem cell (iPSC) model systems have been developed to better understand the molecular roles of MeCP2/MECP2 and gain insight on the disease pathophysiology. Recently, there has been an interest in the determining the glial component of RTT pathology. To model the non-cell autonomous glial component in RTT pathology in a human-based system, we have developed a patient-derived RTT astrocyte/neuron co-culture system. We have validated a detrimental non-cell autonomous effect of RTT astrocytes, and by utilizing three different RTT mutational lines, have determined that this effect is consistent and is mediated in part by astrocyte secreted factors. We have also gone on to test the efficacy of neurotrophic factors in ameliorating this glial effect and have found insulin-like growth factor 1 (IGF-1) and its tripeptide cleavage product, Glycine-Proline-Glutamate (GPE), to increase the somal area and to promote neurite outgrowth in GABAergic interneurons.

Modelling Genetic Heart Diseases with Patient-specific Induced Pluripotent Stem Cells

Modelling Genetic Heart Diseases with Patient-specific Induced Pluripotent Stem Cells
Author:
Publisher:
Total Pages: 115
Release: 2014
Genre:
ISBN:

Brugada syndrome (BrS), a cardiac genetic disease, is one of the major causes of sudden cardiac death in healthy young people. However, the underlying electrophysiological and molecular mechanisms have not been completely understood. A major challenge in cardiac translational research is the lack of tissue culture systems replicating human pathology to study disease mechanisms and to identify druggable targets. Aim of this study was to study the pathophysiological and molecular mechanisms of BrS in vitro using patient-specific induced pluripotent stem cells (iPSCs) as a renewable and unlimi...

Current Progress in iPSC Disease Modeling

Current Progress in iPSC Disease Modeling
Author: Alexander Birbrair
Publisher: Academic Press
Total Pages: 326
Release: 2021-11-11
Genre: Science
ISBN: 0323856438

Current Progress in iPSC Disease Modeling, Volume Fourteen in the Advances in Stem Cell Biology series, is a timely and expansive collection of information and new discoveries in the field. This new volume addresses advances in research on how induced pluripotent stem cells are used for the creation of new tissues and organs. The creation of iPSC technology allowed the development of disease-specific human pluripotent stem cells. These cells allow researchers to study questions once impossible for some human diseases. This volume addresses iPSCs for vascular tissue engineering, bioprinting, derived lung organoids for pulmonary disorders, skeletal muscle engineering, human kidney organoids, and more. It is written for researchers and scientists in stem cell therapy, cell biology, regenerative medicine and organ transplantation, and is contributed by world-renowned authors in the field. Provides an overview of the fast-moving field of stem cell biology and function, regenerative medicine and therapeutics Covers advances in research on how induced pluripotent stem cells are used to create new tissues/organs Contributed by world-renowned experts in the field

Recent Advances in iPSC Disease Modeling

Recent Advances in iPSC Disease Modeling
Author: Alexander Birbrair
Publisher: Academic Press
Total Pages: 322
Release: 2020-07-16
Genre: Science
ISBN: 0128232692

Recent Advances in iPSC Disease Modeling, Volume One addresses how induced pluripotent stem cells can be used to model various diseases. This new volume teaches readers about current advances in the field, describing the use of induced pluripotent stem cells to model several diseases in vitro, and thus enabling us to study the cellular and molecular mechanisms involved in different pathologies. Further insights into these mechanisms will have important implications for our understanding of disease appearance, development and progression. The volume is written for researchers and scientists in stem cell therapy, cell biology, regenerative medicine and organ transplantation specialists. In recent years, remarkable progress has been made in the obtention of induced pluripotent stem cells and their differentiation into several cell types, tissues and organs using state-of-art techniques. Hence, these advantages have facilitated the identification of key targets and further defining on the molecular basis of several disorders. Provides an overview on the fast-moving field of induced pluripotent stem cell technology, regenerative medicine and therapeutics Covers the following diseases: severe congenital neutropenia, sickle cell and Diamond-Blackfan anemias, muscular dystrophies, Bernard-Soulier syndrome, familial hypercholesterolemia type II A, Werner syndrome, lysosomal storage diseases, and more Contains descriptions of cutting-edge research on the development of disease-specific human pluripotent stem cells

Adult Stem Cells

Adult Stem Cells
Author: Paolo Di Nardo
Publisher: Springer Nature
Total Pages: 341
Release:
Genre:
ISBN: 1071639951