Emptying the stores: lysosomal diseases and therapeutic strategies

FM Platt - Nature reviews Drug discovery, 2018 - nature.com
Lysosomal storage disorders (LSDs)—designated as' orphan'diseases—are inborn errors of
metabolism caused by defects in genes that encode proteins involved in various aspects of …

Small heat shock proteins, big impact on protein aggregation in neurodegenerative disease

JM Webster, AL Darling, VN Uversky… - Frontiers in …, 2019 - frontiersin.org
Misfolding, aggregation, and aberrant accumulation of proteins are central components in
the progression of neurodegenerative disease. Cellular molecular chaperone systems …

Modulation of neurodegeneration by molecular chaperones

PJ Muchowski, JL Wacker - Nature Reviews Neuroscience, 2005 - nature.com
Many neurodegenerative disorders are characterized by conformational changes in proteins
that result in misfolding, aggregation and intra-or extra-neuronal accumulation of amyloid …

HSP72 protects against obesity-induced insulin resistance

J Chung, AK Nguyen, DC Henstridge… - Proceedings of the …, 2008 - National Acad Sciences
Patients with type 2 diabetes have reduced gene expression of heat shock protein (HSP) 72,
which correlates with reduced insulin sensitivity. Heat therapy, which activates HSP72 …

Heat shock transcription factor 1 as a therapeutic target in neurodegenerative diseases

DW Neef, AM Jaeger, DJ Thiele - Nature reviews Drug discovery, 2011 - nature.com
Neurodegenerative diseases such as Alzheimer's disease, Parkinson's disease,
Huntington's disease, amyotrophic lateral sclerosis and prion-based neurodegeneration are …

Treatment with arimoclomol, a coinducer of heat shock proteins, delays disease progression in ALS mice

D Kieran, B Kalmar, JRT Dick, J Riddoch-Contreras… - Nature medicine, 2004 - nature.com
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative condition in which
motoneurons of the spinal cord and motor cortex die, resulting in progressive paralysis,. This …

Heat shock response modulators as therapeutic tools for diseases of protein conformation

SD Westerheide, RI Morimoto - Journal of Biological Chemistry, 2005 - ASBMB
The disruption of protein folding quality control results in the accumulation of a non-native
protein species that can form oligomers, aggregates, and inclusions indicative of …

Heat shock proteins in the regulation of apoptosis: new strategies in tumor therapy: a comprehensive review

AS Sreedhar, P Csermely - Pharmacology & therapeutics, 2004 - Elsevier
Heat shock proteins (Hsp) form the most ancient defense system in all living organisms on
earth. These proteins act as molecular chaperones by helping in the refolding of misfolded …

Understanding the phenotypic variability in Niemann-Pick disease type C (NPC): a need for precision medicine

M Las Heras, B Szenfeld, RA Ballout, E Buratti… - NPJ Genomic …, 2023 - nature.com
Abstract Niemann-Pick type C (NPC) disease is a lysosomal storage disease (LSD)
characterized by the buildup of endo-lysosomal cholesterol and glycosphingolipids due to …

Hsp70 and its molecular role in nervous system diseases

G Turturici, G Sconzo, F Geraci - Biochemistry research …, 2011 - Wiley Online Library
Heat shock proteins (HSPs) are induced in response to many injuries including stroke,
neurodegenerative disease, epilepsy, and trauma. The overexpression of one HSP in …