Glycosidase-targeting small molecules for biological and therapeutic applications

Y Kim, H Li, J Choi, J Boo, H Jo, JY Hyun… - Chemical Society …, 2023 - pubs.rsc.org
Glycosidases are ubiquitous enzymes that catalyze the hydrolysis of glycosidic linkages in
oligosaccharides and glycoconjugates. These enzymes play a vital role in a wide variety of …

Enzyme therapy: current challenges and future perspectives

M de la Fuente, L Lombardero… - International Journal of …, 2021 - mdpi.com
In recent years, enzymes have risen as promising therapeutic tools for different pathologies,
from metabolic deficiencies, such as fibrosis conditions, ocular pathologies or joint …

Treatable inherited metabolic disorders causing intellectual disability: 2021 review and digital app

EMM Hoytema van Konijnenburg, SB Wortmann… - Orphanet journal of rare …, 2021 - Springer
Abstract Background The Treatable ID App was created in 2012 as digital tool to improve
early recognition and intervention for treatable inherited metabolic disorders (IMDs) …

Chronic exposure to the star polycation (SPc) nanocarrier in the larval stage adversely impairs life history traits in Drosophila melanogaster

S Yan, N Li, Y Guo, Y Chen, C Ji, M Yin, J Shen… - Journal of …, 2022 - Springer
Background Nanomaterials are widely used as pesticide adjuvants to increase pesticide
efficiency and minimize environmental pollution. But it is increasingly recognized that …

Mechanism of secondary ganglioside and lipid accumulation in lysosomal disease

B Breiden, K Sandhoff - International journal of molecular sciences, 2020 - mdpi.com
Gangliosidoses are caused by monogenic defects of a specific hydrolase or an ancillary
sphingolipid activator protein essential for a specific step in the catabolism of gangliosides …

[HTML][HTML] Models to study basic and applied aspects of lysosomal storage disorders

Á Gaudioso, TP Silva, MD Ledesma - Advanced Drug Delivery Reviews, 2022 - Elsevier
The lack of available treatments and fatal outcome in most lysosomal storage disorders
(LSDs) have spurred research on pathological mechanisms and novel therapies in recent …

Whole exome sequencing in childhood-onset lupus frequently detects single gene etiologies

I Tirosh, S Spielman, O Barel, R Ram, T Stauber… - Pediatric …, 2019 - Springer
Background Systemic lupus erythematosus (SLE) comprise a diverse range of clinical
manifestations. To date, more than 30 single gene causes of lupus/lupus like syndromes in …

Enzyme replacement therapy for genetic disorders associated with enzyme deficiency

M Marchetti, S Faggiano… - Current Medicinal …, 2022 - ingentaconnect.com
Mutations in human genes might lead to the loss of functional proteins, causing diseases.
Among these genetic disorders, a large class is associated with the deficiency in metabolic …

[HTML][HTML] Food and Drug Administration (FDA) Approvals of Biological Drugs in 2023

AC Martins, MY Oshiro, F Albericio, BG de la Torre - Biomedicines, 2024 - mdpi.com
An increase in total drug (small molecules and biologics) approvals by the Food and Drug
Administration (FDA) was seen in 2023 compared with the previous year. Cancer remained …

Targeting cancer via Golgi α-mannosidase II inhibition: How far have we come in developing effective inhibitors?

ZY Lee, JSE Loo, A Wibowo, MF Mohammat… - Carbohydrate …, 2021 - Elsevier
Dysregulation of glycosylation pathways has been well documented in several types of
cancer, where it often participates in cancer development and progression, especially …