Substrate reduction therapy for GBA1-associated Parkinsonism: Are we betting on the wrong mouse?

Mov Disord. 2020 Feb;35(2):228-230. doi: 10.1002/mds.27903. Epub 2019 Nov 11.

Authors

Ellen Sidransky¹, David Arkadir^{2

3}, Peter Bauer⁴, Tama Dinur⁵, Grisel Lopez¹, Arndt Rolfs⁴, Ari Zimran^{3

5}

Affiliations

¹ Medical Genetics Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, Maryland, USA.
² Hadassah Medical Center, Jerusalem, Israel.
³ Hebrew University-Hadassah Medical School, Jerusalem, Israel.
⁴ Centogene AG, Rostock, Germany.
⁵ Gaucher Unit, Shaare Zedek Medical Center, Jerusalem, Israel.

PMID: 31710399
DOI: 10.1002/mds.27903

No abstract available

Keywords: GBA1-associated parkinsonism; Gaucher carriers; Parkinson's disease; glucocerebrosidase; substrate reduction therapy.

Publication types

Research Support, N.I.H., Intramural
Review

MeSH terms

Animals
Disease Models, Animal
Gaucher Disease / metabolism*
Gaucher Disease / physiopathology
Glucosylceramidase / metabolism*
Humans
Mice
Parkinsonian Disorders / metabolism
Parkinsonian Disorders / physiopathology*

Substances

Glucosylceramidase