Substrate reduction therapy for Krabbe disease and metachromatic leukodystrophy using a novel ceramide galactosyltransferase inhibitor
Abstract Krabbe disease (KD) and metachromatic leukodystrophy (MLD) are caused by accumulation of the glycolipids galactosylceramide (GalCer) and sulfatide and their toxic metabolites psychosine and lysosulfatide, respectively. We discovered a potent and selective small molecule inhibitor (S202) of...
Main Authors: | Michael C. Babcock, Christina R. Mikulka, Bing Wang, Sanjay Chandriani, Sundeep Chandra, Yue Xu, Katherine Webster, Ying Feng, Hemanth R. Nelvagal, Alex Giaramita, Bryan K. Yip, Melanie Lo, Xuntian Jiang, Qi Chao, Josh C. Woloszynek, Yuqiao Shen, Shripad Bhagwat, Mark S. Sands, Brett E. Crawford |
---|---|
Format: | Article |
Language: | English |
Published: |
Nature Publishing Group
2021-07-01
|
Series: | Scientific Reports |
Online Access: | https://doi.org/10.1038/s41598-021-93601-1 |
Similar Items
-
Metachromatic Leukodystrophy
by: J Gordon Millichap
Published: (1990-01-01) -
Metachromatic leukodystrophy
by: Prima Cheryl D′souza, et al.
Published: (2013-01-01) -
Metachromatic Leukodystrophy Variants
by: J Gordon Millichap
Published: (1993-08-01) -
Molecular Basis of Metachromatic Leukodystrophies
by: J Gordon Millichap
Published: (1991-02-01) -
Adult metachromatic leukodystrophy: case report
by: T. I. Prusova, et al.
Published: (2021-04-01)