[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 …
and recycling cellular waste, cellular signalling and energy metabolism. Defects in genes …
The rapidly evolving view of lysosomal storage diseases
Lysosomal storage diseases are a group of metabolic disorders caused by deficiencies of
several components of lysosomal function. Most commonly affected are lysosomal …
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
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 …
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 …
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 …
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
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 …
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 …
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 …
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 …
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 …
vehicles for targeted delivery of therapeutic agents to treat difficult-to-manage human …