Lysosomes as coordinators of cellular catabolism, metabolic signalling and organ physiology

C Settembre, RM Perera - Nature Reviews Molecular Cell Biology, 2024 - nature.com
Every cell must satisfy basic requirements for nutrient sensing, utilization and recycling
through macromolecular breakdown to coordinate programmes for growth, repair and stress …

Lysosomes as dynamic regulators of cell and organismal homeostasis

A Ballabio, JS Bonifacino - Nature reviews Molecular cell biology, 2020 - nature.com
Exciting new discoveries have transformed the view of the lysosome from a static organelle
dedicated to the disposal and recycling of cellular waste to a highly dynamic structure that …

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 …

Targeting lysosomal degradation pathways: new strategies and techniques for drug discovery

J Pei, G Wang, L Feng, J Zhang, T Jiang… - Journal of Medicinal …, 2021 - ACS Publications
A series of tools for targeted protein degradation are inspiring scientists to develop new
drugs with advantages over traditional small-molecule drugs. Among these tools, proteolysis …

Current and future prospects for gene therapy for rare genetic diseases affecting the brain and spinal cord

TL Jensen, CR Gøtzsche… - Frontiers in molecular …, 2021 - frontiersin.org
In recent years, gene therapy has been raising hopes toward viable treatment strategies for
rare genetic diseases for which there has been almost exclusively supportive treatment. We …

Membrane trafficking in health and disease

R Yarwood, J Hellicar… - Disease models & …, 2020 - journals.biologists.com
Membrane trafficking pathways are essential for the viability and growth of cells, and play a
major role in the interaction of cells with their environment. In this At a Glance article and …

Ubiquilins regulate autophagic flux through mTOR signalling and lysosomal acidification

M Şentürk, G Lin, Z Zuo, D Mao, E Watson… - Nature cell …, 2019 - nature.com
Although the aetiology of amyotrophic lateral sclerosis (ALS) remains poorly understood,
impaired proteostasis is a common feature of different forms of ALS. Mutations in genes …

Neuronal ceroid lipofuscinosis: the multifaceted approach to the clinical issues, an overview

A Simonati, RE Williams - Frontiers in neurology, 2022 - frontiersin.org
The main aim of this review is to summarize the current state-of-art in the field of childhood
Neuronal Ceroid Lipofuscinosis (NCL), a group of rare neurodegenerative disorders. These …

Built to last: lysosome remodeling and repair in health and disease

R Zoncu, RM Perera - Trends in cell biology, 2022 - cell.com
Lysosomes play major roles in growth regulation and catabolism and are recognized as
critical mediators of cellular remodeling. An emerging theme is how the lysosome is itself …

WFS1 functions in ER export of vesicular cargo proteins in pancreatic β-cells

L Wang, H Liu, X Zhang, E Song, Y Wang, T Xu… - Nature …, 2021 - nature.com
The sorting of soluble secretory proteins from the endoplasmic reticulum (ER) to the Golgi
complex is mediated by coat protein complex II (COPII) vesicles and thought to required …