title: Non-iminosugar glucocerebrosidase small molecule chaperones.
abstract: Small molecule chaperones are a promising therapeutic approach for the Lysosomal Storage Disorders (LSDs). Here, we report the discovery of a new series of non-iminosugar glucocerebrosidase inhibitors with chaperone capacity, and describe their structure activity relationship (SAR), selectivity, cell activity phamacokinetics.
authors.affiliation: NIH Chemical Genomics Center, National Human Genome Research Institute, National Institutes of Health, 9800 Medical Center Drive, Rockville, 20850, MD, USA.
authors.forename: Juan Jose
authors.initials: JJ
authors.lastname: Marugan
authors.forename: Wenwei
authors.initials: W
authors.lastname: Huang
authors.forename: Omid
authors.initials: O
authors.lastname: Motabar
authors.forename: Wei
authors.initials: W
authors.lastname: Zheng
authors.forename: Jingbo
authors.initials: J
authors.lastname: Xiao
authors.forename: Samarjit
authors.initials: S
authors.lastname: Patnaik
authors.forename: Noel
authors.initials: N
authors.lastname: Southall
authors.forename: Wendy
authors.initials: W
authors.lastname: Westbroek
authors.forename: Wendy A
authors.initials: WA
authors.lastname: Lea
authors.forename: Anton
authors.initials: A
authors.lastname: Simeonov
authors.forename: Ehud
authors.initials: E
authors.lastname: Goldin
authors.forename: Maria A
authors.initials: MA
authors.lastname: Debernardi
authors.forename: Ellen
authors.initials: E
authors.lastname: Sidransky