Artificially Induced Pluripotent Stem Cell-Derived Whole-Brain Organoid for Modelling the Pathophysiology of Metachromatic Leukodystrophy and Drug Repurposing

Artificially Induced Pluripotent Stem Cell-Derived Whole-Brain Organoid for Modelling the Pathophysiology of Metachromatic Leukodystrophy and Drug Repurposing

Metachromatic leukodystrophy (MLD) is a rare neurodegenerative disease that results from a deficiency of the lysosomal enzyme arylsulfatase A (ARSA). Worldwide, there are between one in 40,000 and one in 160,000 people living with the disease. While there are currently no effective treatments for ML...

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Bibliographic Details
Main Authors: Sally Esmail, Wayne R. Danter
Format: Article
Language:English
Published: MDPI AG 2021-04-01
Series:Biomedicines
Subjects:
metachromatic leukodystrophy
artificially induced whole-brain organoids
drug repurposing
drug discovery
rare metabolic disorder
Online Access:https://www.mdpi.com/2227-9059/9/4/440

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https://www.mdpi.com/2227-9059/9/4/440

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