Current treatment options and novel nanotechnology-driven enzyme replacement strategies for lysosomal storage disorders
A Del Grosso, G Parlanti, R Mezzena… - Advanced Drug Delivery …, 2022 - Elsevier
Lysosomal storage disorders (LSDs) are a vast group of more than 50 clinically identified
metabolic diseases. They are singly rare, but they affect collectively 1 on 5,000 live births …
metabolic diseases. They are singly rare, but they affect collectively 1 on 5,000 live births …
Role of nanotechnology for enzyme replacement therapy in lysosomal diseases. A focus on Gaucher's disease
L Martín-Banderas, MA Holgado… - Current Medicinal …, 2016 - ingentaconnect.com
Lysosomal storage diseases (LSDs) comprise a group of rare inherited chronic syndromes
that cause deficiency of specific native enzymes within the lysosomes. The macromolecular …
that cause deficiency of specific native enzymes within the lysosomes. The macromolecular …
Treatment of lysosomal storage diseases: recent patents and future strategies
S Ortolano, I Vieitez, C Navarro… - Recent patents on …, 2014 - ingentaconnect.com
Lysosomal storage diseases (LSDs) are a group of rare genetic multisystemic disorders,
resulting in deficient lysosomal activity. These pathologies are characterized by progressive …
resulting in deficient lysosomal activity. These pathologies are characterized by progressive …
Enzyme replacement therapy: a review and its role in treating lysosomal storage diseases
M Li - Pediatric annals, 2018 - journals.healio.com
Lysosomal storage diseases (LSDs) are a heterogeneous group of genetic disorders
caused by defects in lysosomal function that lead to multiorgan system damage. Due to wide …
caused by defects in lysosomal function that lead to multiorgan system damage. Due to wide …
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 …
45 Lysosomal Storage Diseases (LSDs), mostly pre-adult disorders which are associated …
Lysosomal enzyme replacement therapies: Historical development, clinical outcomes, and future perspectives
Lysosomes and lysosomal enzymes play a central role in numerous cellular processes,
including cellular nutrition, recycling, signaling, defense, and cell death. Genetic deficiencies …
including cellular nutrition, recycling, signaling, defense, and cell death. Genetic deficiencies …
Emerging therapies for neuropathic lysosomal storage disorders
JM Kelly, A Bradbury, DR Martin, ME Byrne - Progress in Neurobiology, 2017 - Elsevier
Abstract Approximately 1 in 5000–8000 children are born annually with a lysosomal storage
disease (LSD), which affects their cells' ability to break down naturally occurring substrates …
disease (LSD), which affects their cells' ability to break down naturally occurring substrates …
[HTML][HTML] Enzyme replacement therapies: what is the best option?
Despite many beneficial outcomes of the conventional enzyme replacement therapy (ERT),
several limitations such as the high-cost of the treatment and various inadvertent side effects …
several limitations such as the high-cost of the treatment and various inadvertent side effects …
Treatment perspectives for the lysosomal storage diseases
GA Grabowski - Expert Opinion on Emerging Drugs, 2008 - Taylor & Francis
Background: The therapy of the lysosomal storage diseases (LSDs) was developed by
supplying adequate amounts of the needed enzyme to affected individuals. This approach in …
supplying adequate amounts of the needed enzyme to affected individuals. This approach in …
Treatment options for lysosomal storage disorders: developing insights
CM van Gelder, AAM Vollebregt, I Plug… - Expert opinion on …, 2012 - Taylor & Francis
Introduction: Lysosomal storage disorders (LSDs) are clinically heterogeneous disorders
that result primarily from lysosomal accumulation of macromolecules in various tissues …
that result primarily from lysosomal accumulation of macromolecules in various tissues …