The Experts below are selected from a list of 71013 Experts worldwide ranked by ideXlab platform

Yoon Young Jang - One of the best experts on this subject based on the ideXlab platform.

  • Gene correction in patient-specific iPSCs for therapy development and Disease Modeling
    Human Genetics, 2016
    Co-Authors: Yoon Young Jang, Zhaohui Ye
    Abstract:

    The discovery that mature cells can be reprogrammed to become pluripotent and the development of engineered endonucleases for enhancing genome editing are two of the most exciting and impactful technology advances in modern medicine and science. Human pluripotent stem cells have the potential to establish new model systems for studying human developmental biology and Disease mechanisms. Gene correction in patient-specific iPSCs can also provide a novel source for autologous cell therapy. Although historically challenging, precise genome editing in human iPSCs is becoming more feasible with the development of new genome-editing tools, including ZFNs, TALENs, and CRISPR. iPSCs derived from patients of a variety of Diseases have been edited to correct Disease-associated mutations and to generate isogenic cell lines. After directed differentiation, many of the corrected iPSCs showed restored functionality and demonstrated their potential in cell replacement therapy. Genome-wide analyses of gene-corrected iPSCs have collectively demonstrated a high fidelity of the engineered endonucleases. Remaining challenges in clinical translation of these technologies include maintaining genome integrity of the iPSC clones and the differentiated cells. Given the rapid advances in genome-editing technologies, gene correction is no longer the bottleneck in developing iPSC-based gene and cell therapies; generating functional and transplantable cell types from iPSCs remains the biggest challenge needing to be addressed by the research field.

  • applications of patient specific induced pluripotent stem cells focused on Disease Modeling drug screening and therapeutic potentials for liver Disease
    International Journal of Biological Sciences, 2010
    Co-Authors: Yong Soon Chun, Pooja Chaudhari, Yoon Young Jang
    Abstract:

    The recent advances in the induced pluripotent stem cell (iPSC) research have significantly changed our perspectives on regenerative medicine by providing researchers with a unique tool to derive Disease-specific stem cells for study. In this review, we describe the human iPSC generation from developmentally diverse origins (i.e. endoderm-, mesoderm-, and ectoderm- tissue derived human iPSCs) and multistage hepatic differentiation protocols, and discuss both basic and clinical applications of these cells including Disease Modeling, drug toxicity screening/drug discovery, gene therapy and cell replacement therapy.

Hans Clevers - One of the best experts on this subject based on the ideXlab platform.

  • Disease Modeling in stem cell derived 3d organoid systems
    Trends in Molecular Medicine, 2017
    Co-Authors: Devanjali Dutta, Hans Clevers
    Abstract:

    Organoids are 3D in vitro culture systems derived from self-organizing stem cells. They can recapitulate the in vivo architecture, functionality, and genetic signature of original tissues. Thus, organoid technology has been rapidly applied to understanding stem cell biology, organogenesis, and various human pathologies. The recent development of human patient-derived organoids has enabled Disease Modeling with precision, highlighting their great potential in biomedical applications, translational medicine, and personalized therapy. In light of recent breakthroughs using organoids, it is only apt that we appreciate the advantages and shortcomings of this technology to exploit its full potential. We discuss recent advances in the application of organoids in studying cancer and hereditary Diseases, as well as in the examination of host cell–microorganism interactions.

George Q Daley - One of the best experts on this subject based on the ideXlab platform.

  • 3177 the large scale generation of mature hemoglobinized red blood cells in vitro from human pluripotent stem cells for Disease Modeling and autologous therapies
    Experimental Hematology, 2019
    Co-Authors: Ashlee J Conway, Tolulope Roswano, Thomas Williamson, Martha Clarke, Melissa A Kinney, Trista E North, George Q Daley
    Abstract:

    Human induced pluripotent stem cells (iPSC) are an invaluable resource in tissue and blood cell engineering due to their multi-lineage potential in culture systems. iPSC-derived progenitors that undergo induced erythropoiesis in vitro would allow for the Modeling of blood Diseases, such as Sickle Cell Anemia (SCA), for therapeutic applications, as well as the possibility of becoming an autologous source of blood products for patients. The robust generation of terminal, mature red blood cells (RBCs) in large numbers from iPSCs has historically been challenging, due to a lack of terminal erythropoietic development. Here, we describe an optimized method of generating terminal RBCs in vitro from healthy and Sickle homozygous patient-derived iPSCs using a plasma-rich culture media and hypoxic conditioning. Committed erythroid cells underwent prominent proliferation from an enriched CD34+ population (90-fold amplification), as well as efficient adult globin switching (>40%), and enhanced rates of enucleation (>60%). In their terminal state, RBCs with the Sickle homozygous mutation displayed morphological and pathological characteristics that matched the human condition, demonstrating their usefulness in Disease Modeling and translational research. These studies describe an efficient protocol for the generation of committed erythroid cells from pluripotent human iPSCs that recapitulate human blood Diseases in vitro. Ongoing optimization of erythroid culture systems will hopefully drive future development of autologous cell therapies for patients with rare blood types.

  • reprogrammed cells for Disease Modeling and regenerative medicine
    Annual Review of Medicine, 2013
    Co-Authors: Anne Cherry, George Q Daley
    Abstract:

    The conversion of somatic cells into pluripotent cells is transforming the way Diseases are researched and treated. Induced pluripotent stem (iPS) cells' promise may soon be realized in the field of hematology, as hematopoietic stem cell transplants are already commonplace in clinics around the world. We provide a current comparison between induced pluripotent and embryonic stem cells, describe progress toward Modeling hematological disorders using iPS cells, and illustrate the hurdles that must be overcome before iPS cell therapies will be available in clinics.

Jack M. Parent - One of the best experts on this subject based on the ideXlab platform.

  • Using Patient-Derived Induced Pluripotent Stem Cells to Model and Treat Epilepsies
    Current Neurology and Neuroscience Reports, 2015
    Co-Authors: Xixi Du, Jack M. Parent
    Abstract:

    Human induced pluripotent stem cells (iPSCs) are transforming the fields of Disease Modeling and precision therapy. For the treatment of neurological disorders, iPSCs introduce the possibility for targeted cell-based therapies by deriving patient-specific neural tissue in vitro that may ultimately be used for transplantation. We review iPSC technologies and their applications that have already advanced our understanding of neurological disorders, focusing on the epilepsies. We also discuss the application of powerful new tools such as genome editing and multi-well, multi-electrode array recording platforms to iPSC Disease Modeling and therapy development for the epilepsies. Despite some limitations, the field of iPSCs is evolving rapidly and is quickly becoming vital for understanding mechanisms of genetic epilepsies and for future patient-specific therapeutic applications.

Joseph C Wu - One of the best experts on this subject based on the ideXlab platform.

  • induced pluripotent stem cell derived cardiomyocytes for cardiovascular Disease Modeling and drug screening
    Stem Cell Research & Therapy, 2013
    Co-Authors: Arun Sharma, Joseph C Wu, Sean M Wu
    Abstract:

    Human induced pluripotent stem cells (hiPSCs) have emerged as a novel tool for drug discovery and therapy in cardiovascular medicine. hiPSCs are functionally similar to human embryonic stem cells (hESCs) and can be derived autologously without the ethical challenges associated with hESCs. Given the limited regenerative capacity of the human heart following myocardial injury, cardiomyocytes derived from hiPSCs (hiPSC-CMs) have garnered significant attention from basic and translational scientists as a promising cell source for replacement therapy. However, ongoing issues such as cell immaturity, scale of production, inter-line variability, and cell purity will need to be resolved before human clinical trials can begin. Meanwhile, the use of hiPSCs to explore cellular mechanisms of cardiovascular Diseases in vitro has proven to be extremely valuable. For example, hiPSC-CMs have been shown to recapitulate Disease phenotypes from patients with monogenic cardiovascular disorders. Furthermore, patient-derived hiPSC-CMs are now providing new insights regarding drug efficacy and toxicity. This review will highlight recent advances in utilizing hiPSC-CMs for cardiac Disease Modeling in vitro and as a platform for drug validation. The advantages and disadvantages of using hiPSC-CMs for drug screening purposes will be explored as well.

  • induced pluripotent stem cells as a Disease Modeling and drug screening platform
    Journal of Cardiovascular Pharmacology, 2012
    Co-Authors: Antje D Ebert, Ping Liang, Joseph C Wu
    Abstract:

    Induced pluripotent stem cells (iPSCs) hold great hopes for therapeutic application in various Diseases. While ongoing research is dedicated to achieving clinical translation of iPSCs, further understanding of the mechanisms that underlie complex pathogenic conditions is required. Compared to other classical models for studying Diseases, iPSCs provide considerable advantages. A newly emerging application of iPSCs is in vitro Disease Modeling, which can significantly improve the never-ending search for new pharmacological cures. Here, we will discuss current efforts to create iPSC-dependent, patient-specific Disease models. Furthermore, we will review the use of iPSCs for development and testing of new therapeutic agents, and the implications for high-throughput drug screening.