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Lysosomal Enzyme Replacement Therapies: Historical Development, Clinical Outcomes, And Future Perspectives
M. Solomon, S. Muro
Published 2017 · Medicine
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ABSTRACT Lysosomes and lysosomal enzymes play a central role in numerous cellular processes, including cellular nutrition, recycling, signaling, defense, and cell death. Genetic deficiencies of lysosomal components, most commonly enzymes, are known as “lysosomal storage disorders” or “lysosomal diseases” (LDs) and lead to lysosomal dysfunction. LDs broadly affect peripheral organs and the central nervous system (CNS), debilitating patients and frequently causing fatality. Among other approaches, enzyme replacement therapy (ERT) has advanced to the clinic and represents a beneficial strategy for 8 out of the 50–60 known LDs. However, despite its value, current ERT suffers from several shortcomings, including various side effects, development of “resistance”, and suboptimal delivery throughout the body, particularly to the CNS, lowering the therapeutic outcome and precluding the use of this strategy for a majority of LDs. This review offers an overview of the biomedical causes of LDs, their socio‐medical relevance, treatment modalities and caveats, experimental alternatives, and future treatment perspectives. Graphical abstract Figure. No Caption available.
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Large Biomolecules: An Overview
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Alpha-Mannosidosis: Therapeutic Strategies
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Pharmacological Chaperones for the Mucopolysaccharidoses
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Enzyme prodrug therapies and therapeutic enzymes.
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Advances in the treatment of neuronal ceroid lipofuscinosis
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The Place for Enzymes and Biologically Active Peptides from Marine Organisms for Application in Industrial and Pharmaceutical Biotechnology.
Jean-Étienne R L Morlighem (2019)
New Advanced Strategies for the Treatment of Lysosomal Diseases Affecting the Central Nervous System.
M. R. Gigliobianco (2019)
Albumin enhances dextran NP’s delivery and therapeutic efficacy of PTX for colorectal cancer
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Advances in the Development of Pharmacological Chaperones for the Mucopolysaccharidoses
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Strategies for Enhancing the Permeation of CNS-Active Drugs through the Blood-Brain Barrier: A Review
Isra' Zeiadeh (2018)
Gene therapy for neurological disorders: challenges and recent advancements
Stefanie A Pena (2019)
Application of advances in endocytosis and membrane trafficking to drug delivery.
Yaping Ju (2020)
Diagnosis, prognosis, and treatment of leukodystrophies
M. S. Knaap (2019)
Peripheral neuropathy in metachromatic leukodystrophy: current status and future perspective
Shanice Beerepoot (2019)
δ-Tocopherol Effect on Endocytosis and Its Combination with Enzyme Replacement Therapy for Lysosomal Disorders: A New Type of Drug Interaction?
R. L. Manthe (2019)
Role of induced pluripotent stem cells in lysosomal storage diseases
J. Kido (2020)
RareLSD: a manually curated database of lysosomal enzymes associated with rare diseases
Sana Akhter (2019)
Pharmacokinetics and pharmacodynamics of PEGylated truncated human cystathionine beta‐synthase for treatment of homocystinuria
Tomas Majtan (2018)
Current strategies for the treatment of inborn errors of metabolism.
M. Gambello (2018)
Neuronal Ceroid Lipofuscinosis: Potential for Targeted Therapy
N. Specchio (2020)
Gaucher’s Disease and Hurler’s Syndrome in Two First Cousins
Patricia Fenton-Navarro (2017)
A perspective on research, diagnosis, and management of lysosomal storage disorders in Colombia
María Alejandra Puentes-Tellez (2020)
Utility of rapid whole-exome sequencing in the diagnosis of Niemann–Pick disease type C presenting with fetal hydrops and acute liver failure
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