Role of induced pluripotent stem cells in lysosomal storage diseases
نویسندگان
چکیده
منابع مشابه
Induced pluripotent stem cell models of lysosomal storage disorders
Induced pluripotent stem cells (iPSCs) have provided new opportunities to explore the cell biology and pathophysiology of human diseases, and the lysosomal storage disorder research community has been quick to adopt this technology. Patient-derived iPSC models have been generated for a number of lysosomal storage disorders, including Gaucher disease, Pompe disease, Fabry disease, metachromatic ...
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Regenerative capacity of mammals is limited and can rarely regenerate a specific organ or tissue fully. Due to these limitations, regenerative medicine seeks efficient and safe cell sources for regeneration of damaged tissues and organs or treatment for incurable diseases. Human embryonic stem cells (HESCs) hold two important properties called self renewal and pluripotency. However, the use of ...
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Most lysosomal storage diseases (LSDs) are life-threatening genetic diseases. The pathogenesis of these diseases is poorly understood. Induced pluripotent stem (iPS) cell technology offers new opportunities for both mechanistic studies and development of stem cell- based therapies. Here we report the generation of disease-specific iPS cells from mouse models of Fabry disease, globoid cell leuko...
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Human embryonic stem cells (hESCs) were first established as an in vitro culture system (Thomson et al., 1998). hESCs are pluripotent cells that are able to self-renew and can differentiate into the three primary germ layers: endoderm, ectoderm and mesoderm. Specifically, hESCs can be used to generate gut epithelium, cartilage, bone, muscle, neuroepithelium, and embryonic ganglia (Zhang et al.,...
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ژورنال
عنوان ژورنال: Molecular and Cellular Neuroscience
سال: 2020
ISSN: 1044-7431
DOI: 10.1016/j.mcn.2020.103540