Targeted transport of biotherapeutics at the blood-brain barrier

T Moos, MS Thomsen, A Burkhart, E Hede… - Expert opinion on drug …, 2023 - Taylor & Francis
Introduction The treatment of neurological diseases is significantly hampered by the lack of
available therapeutics. A major restraint for the development of drugs is denoted by the …

Gaucher disease provides a unique window into Parkinson disease pathogenesis

E Hertz, Y Chen, E Sidransky - Nature Reviews Neurology, 2024 - nature.com
An exciting development in the field of neurodegeneration is the association between the
rare monogenic disorder Gaucher disease and the common complex disorder Parkinson …

A Fixable Fluorescence‐Quenched Substrate for Quantitation of Lysosomal Glucocerebrosidase Activity in Both Live and Fixed Cells

S Zhu, MC Deen, Y Zhu, PA Gilormini… - Angewandte …, 2023 - Wiley Online Library
Fluorogenic substrates are emerging tools that enable studying enzymatic processes within
their native cellular environments. However, fluorogenic substrates that function within live …

Blood–brain barrier alterations and their impact on Parkinson's disease pathogenesis and therapy

K Lau, R Kotzur, F Richter - Translational Neurodegeneration, 2024 - Springer
There is increasing evidence for blood–brain barrier (BBB) alterations in Parkinson's
disease (PD), the second most common neurodegenerative disorder with rapidly rising …

Characterization of Novel Human β-glucocerebrosidase Antibodies for Parkinson's Disease Research

T Jong, A Gehrlein, E Sidransky… - Journal of …, 2024 - content.iospress.com
Background: Mutations in GBA1, which encodes the lysosome enzyme ß-
glucocerebrosidase (also referred to as acid ßglucosidase or GCase), are the most common …

The SATB1‐MIR22‐GBA axis mediates glucocerebroside accumulation inducing a cellular senescence‐like phenotype in dopaminergic neurons

T Russo, B Kolisnyk, A BS, J Plessis‐Belair, TW Kim… - Aging cell, 2024 - Wiley Online Library
Idiopathic Parkinson's disease (PD) is characterized by the loss of dopaminergic neurons in
the substantia nigra pars compacta, which is associated with neuroinflammation and …

A plug-and-play monofunctional platform for targeted degradation of extracellular proteins and vesicles

S Yao, Y Wang, Q Tang, Y Yin, Y Geng, L Xu… - Nature …, 2024 - nature.com
Existing strategies use bifunctional chimaeras to mediate extracellular protein degradation.
However, these strategies rely on specific lysosome-trafficking receptors to facilitate …

[HTML][HTML] Targeting dysregulated lipid metabolism for the treatment of Alzheimer's disease and Parkinson's disease: Current advancements and future prospects

B Tong, Y Ba, Z Li, C Yang, K Su, H Qi, D Zhang… - Neurobiology of …, 2024 - Elsevier
Alzheimer's and Parkinson's diseases are two of the most frequent neurological diseases.
The clinical features of AD are memory decline and cognitive dysfunction, while PD mainly …

High-throughput screening for small-molecule stabilizers of misfolded glucocerebrosidase in Gaucher disease and Parkinson's disease

D Williams, LM Glasstetter, TT Jong, T Chen… - Proceedings of the …, 2024 - pnas.org
Glucocerebrosidase (GCase) is implicated in both a rare, monogenic disorder (Gaucher
disease, GD) and a common, multifactorial condition (Parkinson's disease, PD); hence, it is …

Enhanced in vivo blood brain barrier transcytosis of macromolecular cargo using an engineered pH-sensitive mouse transferrin receptor binding nanobody

TJ Esparza, S Su, CM Francescutti… - Fluids and Barriers of …, 2023 - Springer
Background The blood brain barrier limits entry of macromolecular diagnostic and
therapeutic cargos. Blood brain barrier transcytosis via receptor mediated transport systems …