[HTML][HTML] Thematic Review Series: Recent Advances in the Treatment of Lysosomal Storage Diseases: Lysosomal storage diseases and the heat shock response …

L Ingemann, T Kirkegaard - Journal of Lipid Research, 2014 - ncbi.nlm.nih.gov
Lysosomes play a vital role in the maintenance of cellular homeostasis through the recycling
of cell constituents, a key metabolic function which is highly dependent on the correct …

[HTML][HTML] Lysosomal storage diseases and the heat shock response: convergences and therapeutic opportunities

L Ingemann, T Kirkegaard - Journal of lipid research, 2014 - ASBMB
Lysosomes play a vital role in the maintenance of cellular homeostasis through the recycling
of cell constituents, a key metabolic function which is highly dependent on the correct …

Modulating the heat-shock response: a potential therapy for lysosomal storage disorders

J Gray - 2014 - ora.ox.ac.uk
Lysosomal storage disorders (LSDs) comprise a broad disease group of inherited metabolic
disorders, the majority of which are associated with CNS pathology, significant disability and …

HSP70 and lysosomal storage disorders: novel therapeutic opportunities

NHT Petersen, T Kirkegaard - 2010 - portlandpress.com
Lysosomes, with their arsenal of catabolic enzymes and crucial metabolic housekeeping
functions are experiencing a revived research interest after having lived a rather quiet life for …

Emerging therapies and therapeutic concepts for lysosomal storage diseases

T Kirkegaard - Expert Opinion on Orphan Drugs, 2013 - Taylor & Francis
Introduction: The success of the first enzyme replacement therapy (ERT) for a lysosomal
storage disease (LSD) and the regulatory and commercial incentives provided by authorities …

Therapies for lysosomal storage diseases: Principles, practice, and prospects for refinements based on evolving science

GA Grabowski, PK Mistry - Molecular Genetics and Metabolism, 2022 - Elsevier
The past three decades of enzyme therapy (ET) for lysosomal storage diseases (LSDs) have
been life-saving and have improved lives of many affected individuals and their families. The …

Editorial [Hot Topic: Recent Advances in Treatment Approaches of Lysosomal Storage Diseases (LSDs)(Guest Editor: Paula Rozenfeld)]

P Rozenfeld - Current Pharmaceutical Biotechnology, 2011 - ingentaconnect.com
This special topic issue published in the journal Current Pharmaceutical Biotechnology is
devoted to Recent advances in treatment approaches of lysosomal storage diseases …

Damage Mediated by Dysfunction of Organelles and Cellular Systems: Lysosomes

M Marí, JC Fernández-Checa - 2014 - digital.csic.es
Lysosomes were discovered more than five decades ago and characterized as cytoplasmic
organelles loaded with acid hydrolases responsible for the digestion of macromolecules and …

Recent developments in therapeutic approaches for lysosomal storage diseases

L Urbanelli, A Magini, A Polchi… - Recent Patents on …, 2011 - ingentaconnect.com
Genetic mutations that cause specific lysosomal protein deficiencies account for more than
45 Lysosomal Storage Diseases (LSDs), mostly pre-adult disorders which are associated …

The blood–brain barrier and treatment of lysosomal storage diseases

JM Aerts, MJ Van Breemen, AP Bussink… - International Congress …, 2005 - Elsevier
Lysosomes are ubiquitous, acidic intracellular compartments that fulfil a crucial role in
degradation of macromolecules in cells. Specific lysosomal catabolic reactions may be …