Human iPSC-Based Models for the Development of Therapeutics Targeting Neurodegenerative Lysosomal Storage Diseases

Human iPSC-Based Models for the Development of Therapeutics Targeting Neurodegenerative Lysosomal Storage Diseases

Lysosomal storage diseases (LSDs) are a group of rare genetic conditions. The absence or deficiency of lysosomal proteins leads to excessive storage of undigested materials and drives secondary pathological mechanisms including autophagy, calcium homeostasis, ER stress, and mitochondrial abnormaliti...

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Bibliographic Details
Main Authors: Marco Luciani, Angela Gritti, Vasco Meneghini
Format: Article
Language:English
Published: Frontiers Media S.A. 2020-09-01
Series:Frontiers in Molecular Biosciences
Subjects:
lysosomal storage disorders
central nervous system
induced pluripotent stem cells
neural stem cells
organoids
drug discovery
Online Access:https://www.frontiersin.org/article/10.3389/fmolb.2020.00224/full

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https://www.frontiersin.org/article/10.3389/fmolb.2020.00224/full

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