[HTML][HTML] Lysosomes as a therapeutic target

SR Bonam, F Wang, S Muller - Nature reviews Drug discovery, 2019 - nature.com
Lysosomes are membrane-bound organelles with roles in processes involved in degrading
and recycling cellular waste, cellular signalling and energy metabolism. Defects in genes …

The rapidly evolving view of lysosomal storage diseases

G Parenti, DL Medina, A Ballabio - EMBO molecular medicine, 2021 - embopress.org
Lysosomal storage diseases are a group of metabolic disorders caused by deficiencies of
several components of lysosomal function. Most commonly affected are lysosomal …

[HTML][HTML] Mechanistic understanding of the toxic effects of arsenic and warfare arsenicals on human health and environment

S Muzaffar, J Khan, R Srivastava, MS Gorbatyuk… - Cell Biology and …, 2023 - Springer
Worldwide, more than 200 million people are estimated to be exposed to unsafe levels of
arsenic. Chronic exposure to unsafe levels of groundwater arsenic is responsible for …

[HTML][HTML] Structure of human lysosomal acid α-glucosidase–a guide for the treatment of Pompe disease

V Roig-Zamboni, B Cobucci-Ponzano, R Iacono… - Nature …, 2017 - nature.com
Pompe disease, a rare lysosomal storage disease caused by deficiency of the lysosomal
acid α-glucosidase (GAA), is characterized by glycogen accumulation, triggering severe …

Transcription factor EB (TFEB) is a new therapeutic target for Pompe disease

C Spampanato, E Feeney, L Li, M Cardone… - EMBO molecular …, 2013 - embopress.org
A recently proposed therapeutic approach for lysosomal storage disorders (LSDs) relies
upon the ability of transcription factor EB (TFEB) to stimulate autophagy and induce …

[HTML][HTML] Pharmacological chaperone therapy: preclinical development, clinical translation, and prospects for the treatment of lysosomal storage disorders

G Parenti, G Andria, KJ Valenzano - Molecular Therapy, 2015 - cell.com
Lysosomal storage disorders (LSDs) are a group of inborn metabolic diseases caused by
mutations in genes that encode proteins involved in different lysosomal functions, in most …

[HTML][HTML] Pompe disease: from pathophysiology to therapy and back again

JA Lim, L Li, N Raben - Frontiers in aging neuroscience, 2014 - frontiersin.org
Pompe disease is a lysosomal storage disorder in which acid alpha-glucosidase (GAA) is
deficient or absent. Deficiency of this lysosomal enzyme results in progressive expansion of …

In vitro and in vivo inhibitory effect of anthocyanin-rich bilberry extract on α-glucosidase and α-amylase

Y Ji, D Liu, J Zhao, J Zhao, H Li, L Li, H Zhang, H Wang - Lwt, 2021 - Elsevier
The inhibition of glycosidase activity is regarded as an effective treatment for diabetes
mellitus. Bilberry (Vaccinium myrtillus L.) fruit has a high content of anthocyanins with …

Inhibitory mechanism of two allosteric inhibitors, oleanolic acid and ursolic acid on α-glucosidase

H Ding, X Hu, X Xu, G Zhang, D Gong - International journal of biological …, 2018 - Elsevier
Glycemic control which can be efficaciously regulated by inhibiting α-glucosidase activity is
an effective therapy for diabetes mellitus. This work is to investigate the kinetics and …

Exosome-and extracellular vesicle-based approaches for the treatment of lysosomal storage disorders

B Lu, J Ku, R Flojo, C Olson, D Bengford… - Advanced Drug Delivery …, 2022 - Elsevier
Cell-generated extracellular vesicles (EVs) are being engineered as biologically-inspired
vehicles for targeted delivery of therapeutic agents to treat difficult-to-manage human …