Alexandra Gehrlein
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View article: Development of quantitative high-throughput screening assays to identify, validate, and optimize small-molecule stabilizers of misfolded β-glucocerebrosidase with therapeutic potential for Gaucher disease and Parkinson’s disease
Development of quantitative high-throughput screening assays to identify, validate, and optimize small-molecule stabilizers of misfolded β-glucocerebrosidase with therapeutic potential for Gaucher disease and Parkinson’s disease Open
Glucocerebrosidase (GCase) is implicated in both a rare, monogenic disorder (Gaucher disease, GD) and a common, multifactorial condition (Parkinson’s disease); hence, it is an urgent therapeutic target. To identify correctors of severe pro…
View article: Characterization of Novel Human β-glucocerebrosidase Antibodies for Parkinson’s Disease Research
Characterization of Novel Human β-glucocerebrosidase Antibodies for Parkinson’s Disease Research Open
Background: Mutations in GBA1, which encodes the lysosome enzyme β-glucocerebrosidase (also referred to as acid β-glucosidase or GCase), are the most common genetic risk factor for Parkinson’s disease (PD) and dementia with Lewy bodies (DL…
View article: Characterization of Novel Human β-glucocerebrosidase Antibodies for Parkinson Disease Research
Characterization of Novel Human β-glucocerebrosidase Antibodies for Parkinson Disease Research Open
BACKGROUND Mutations in GBA1 , which encodes the lysosome enzyme β-glucocerebrosidase (also referred to as acid β-glucosidase or GCase), are the most common genetic risk factor for Parkinson disease (PD) and dementia with Lewy bodies (DLB)…
View article: Targeting neuronal lysosomal dysfunction caused by β-glucocerebrosidase deficiency with an enzyme-based brain shuttle construct
Targeting neuronal lysosomal dysfunction caused by β-glucocerebrosidase deficiency with an enzyme-based brain shuttle construct Open
View article: Targeting neuronal lysosomal dysfunction caused by β-glucocerebrosidase deficiency with an enzyme-based Brain Shuttle construct
Targeting neuronal lysosomal dysfunction caused by β-glucocerebrosidase deficiency with an enzyme-based Brain Shuttle construct Open
Biallelic mutations in GBA1 that lead to reduced β-glucocerebrosidase (GCase) activity result in the monogenic lysosomal storage disease Gaucher disease (GD). Variants in one GBA1 allele are the most common genetic risk factor for multiple…