Tackling neurodegenerative diseases with genomic engineering: A new stem cell initiative from the NIH

Neuron ◽  
2021 ◽  
Vol 109 (7) ◽  
pp. 1080-1083
Author(s):  
Daniel M. Ramos ◽  
William C. Skarnes ◽  
Andrew B. Singleton ◽  
Mark R. Cookson ◽  
Michael E. Ward
Keyword(s):  
Stem Cell ◽  
Neurodegenerative Diseases ◽  
Genomic Engineering

scholarly journals Human iPSC-derived brain endothelial microvessels in a multi-well format enable permeability screens of anti-inflammatory drugs

2021 ◽  
Author(s):  
Sven Fengler ◽  
Birgit Kurkowsky ◽  
Sanjeev Kumar Kaushalya ◽  
Wera Roth ◽  
Philip Denner ◽  
...  
Keyword(s):  
Stem Cell ◽  
Neurodegenerative Diseases ◽  
Induced Pluripotent Stem Cell ◽  
Barrier Properties ◽  
Brain Diseases ◽  
Rapid Screening ◽  
Sodium Fluorescein ◽  
Inflammatory Drugs ◽  
Anti Inflammatory

Optimizing drug candidates for blood-brain barrier (BBB) penetration in humans remains one of the key challenges and many devastating brain diseases including neurodegenerative diseases still do not have adequate treatments. So far, it has been difficult to establish state-of-the-art human stem cell derived in vitro models that mimic physiological barrier properties including a 3D microvasculature in a format that is scalable enough to screen drugs for BBB penetration in early drug development phases. To address this challenge, we established human induced pluripotent stem cell (iPSC)-derived brain endothelial microvessels in a standardized and scalable multi-well plate format. iPSC-derived brain microvascular endothelial cells (BMECs) were supplemented with primary cell conditioned media and grew to intact microvessels in 10 days of culturing. Produced microvessels show a typical BBB phenotype including endothelial protein expression, tight-junctions and polarized localization of efflux transporter. Microvessels exhibited physiological relevant trans-endothelial electrical resistance (TEER), were leak tight for 10 kDa dextran-Alexa 647 and strongly limited the permeability of sodium fluorescein (NaF). Permeability tests with reference compounds confirmed the suitability of our model as platform to identify potential BBB penetrating anti-inflammatory drugs. In summary, the here presented brain microvessel platform recapitulates physiological properties and allows rapid screening of BBB permeable anti-inflammatory compounds that has been suggested as promising substances to cure so far untreatable neurodegenerative diseases.

scholarly journals Microglia alterations in neurodegenerative diseases and their modeling with human induced pluripotent stem cell and other platforms

2020 ◽  
Vol 190 ◽  
pp. 101805 ◽  
Author(s):  
Angélica María Sabogal-Guáqueta ◽  
Alejandro Marmolejo-Garza ◽  
Vítor Passos de Pádua ◽  
Bart Eggen ◽  
Erik Boddeke ◽  
...  
Keyword(s):  
Stem Cell ◽  
Neurodegenerative Diseases ◽  
Pluripotent Stem Cell ◽  
Induced Pluripotent Stem Cell ◽  
Induced Pluripotent

scholarly journals Stem Cell-Derived Exosomes as Therapeutic Approach for Neurodegenerative Disorders: From Biology to Biotechnology

Cells ◽  
2020 ◽  
Vol 9 (12) ◽  
pp. 2663
Author(s):  
Rodrigo Pinheiro Araldi ◽  
Fernanda D’Amelio ◽  
Hugo Vigerelli ◽  
Thatiana Correa de Melo ◽  
Irina Kerkis
Keyword(s):  
Stem Cell ◽  
Cell Therapy ◽  
Neurodegenerative Diseases ◽  
Neurodegenerative Disorders ◽  
Therapeutic Approach ◽  
Rapid Rise ◽  
Cell Based Therapy ◽  
New Treatments ◽  
Medical Advances ◽  
Minimal Information

The aging population has contributed to the rapid rise in the global incidence of neurodegenerative diseases. Despite the medical advances, there are no effective treatments for these disorders. Therefore, there is an urgent need for new treatments for these diseases. In this sense, cell therapy has been recognized as the best candidate for treating incurable diseases, such as neurodegenerative disorders. However, the therapeutic use of these cells can be limited by several factors. Thus, there has been a rediscovery that extracellular vesicles, including exosomes, can be alternatively explored in the treatment of these diseases, overcoming the limits of cell-based therapy. In this sense, this review aims to revisit all areas from biology, including biogenesis and the content of exosomes, to biotechnology, proposing the minimal information required to isolate, characterize, and study the content of these vesicles for scientific and/or clinical purposes.

The Therapeutic Potential of Mesenchymal Stem Cell–Derived Exosomes in Treatment of Neurodegenerative Diseases

2019 ◽  
Vol 56 (12) ◽  
pp. 8157-8167 ◽  
Author(s):  
Armita Mahdavi Gorabi ◽  
Nasim Kiaie ◽  
George E. Barreto ◽  
Morgayn I. Read ◽  
Hossein Ahmadi Tafti ◽  
...  
Keyword(s):  
Stem Cell ◽  
Mesenchymal Stem Cell ◽  
Neurodegenerative Diseases ◽  
Therapeutic Potential

Toward stem cell-based phenotypic screens for neurodegenerative diseases

2015 ◽  
Vol 11 (6) ◽  
pp. 339-350 ◽  
Author(s):  
Vikram Khurana ◽  
Daniel F. Tardiff ◽  
Chee Yeun Chung ◽  
Susan Lindquist
Keyword(s):  
Stem Cell ◽  
Neurodegenerative Diseases

scholarly journals Stem cell technology for neurodegenerative diseases

2011 ◽  
Vol 70 (3) ◽  
pp. 353-361 ◽  
Author(s):  
J. Simon Lunn ◽  
Stacey A. Sakowski ◽  
Junguk Hur ◽  
Eva L. Feldman
Keyword(s):  
Stem Cell ◽  
Neurodegenerative Diseases ◽  
Stem Cell Technology ◽  
Cell Technology
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