ReviewWiley interdisciplinary reviews. Nanomedicine and nanobiotechnology
New biotechnological and nanomedicine strategies for treatment of lysosomal storage disorders.
Review in Wiley interdisciplinary reviews. Nanomedicine and nanobiotechnology. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 43 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Who cites it
43 citing papers in PubMed.
- Investigating the Cellular Effects of GALC Dosing in Enzyme Replacement Therapy for Krabbe Disease Supports the Role of Nanomedicine.Advanced biology · 2025Article
- Therapeutic delivery of recombinant glucocerebrosidase enzyme-containing extracellular vesicles to human cells from Gaucher disease patients.Orphanet journal of rare diseases · 2024Article
- Article
- How Precise are Nanomedicines in Overcoming the Blood-Brain Barrier? A Comprehensive Review of the Literature.International journal of nanomedicine · 2024Review
- Polymer-based drug delivery systems under investigation for enzyme replacement and other therapies of lysosomal storage disorders.Advanced drug delivery reviews · 2023Review
- Targeting the central nervous system in lysosomal storage diseases: Strategies to deliver therapeutics across the blood-brain barrier.Molecular therapy : the journal of the American Society of Gene Therapy · 2023Review
- TweenNanomaterials (Basel, Switzerland) · 2021Article
- Extracellular vesicles from recombinant cell factories improve the activity and efficacy of enzymes defective in lysosomal storage disorders.Journal of extracellular vesicles · 2021Article
- Enzyme Stability in Nanoparticle Preparations Part 1: Bovine Serum Albumin Improves Enzyme Function.Molecules (Basel, Switzerland) · 2020Article
- Mucopolysaccharidosis IVA: Diagnosis, Treatment, and Management.International journal of molecular sciences · 2020Review
- Application of advances in endocytosis and membrane trafficking to drug delivery.Advanced drug delivery reviews · 2020Review
- Brain-targeted enzyme-loaded nanoparticles: A breach through the blood-brain barrier for enzyme replacement therapy in Krabbe disease.Science advances · 2019Article
- Article
- Targeted enzyme delivery systems in lysosomal disorders: an innovative form of therapy for mucopolysaccharidosis.Cellular and molecular life sciences : CMLS · 2019Review
- Biocompatible Polymer Nanoparticles for Drug Delivery Applications in Cancer and Neurodegenerative Disorder Therapies.Journal of functional biomaterials · 2019Review
- Production and Purification of Therapeutic Enzymes.Advances in experimental medicine and biology · 2019Review
- Alterations in Cellular Processes Involving Vesicular Trafficking and Implications in Drug Delivery.Biomimetics (Basel, Switzerland) · 2018Review
- Matrix Metalloproteinase-9-Responsive Nanogels for Proximal Surface Conversion and Activated Cellular Uptake.Biomacromolecules · 2018Article
- Enzyme replacement therapies: what is the best option?BioImpacts : BI · 2018Article
- Effect of temperature on lysosomal enzyme activity during preparation and storage of dried blood spots.Journal of clinical laboratory analysis · 2018Article
Corrections and comments
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Authors and funding
1 author.
Funding
Abstract
This review discusses the multiple bio- and nanotechnological strategies developed in the last few decades for treatment of a group of fatal genetic diseases termed lysosomal storage disorders. Some basic foundation on the biomedical causes and social and clinical relevance of these diseases is provided. Several treatment modalities, from those currently available to novel therapeutic approaches under development, are also discussed; these include gene and cell therapies, substrate reduction therapy, chemical chaperones, enzyme replacement therapy, multifunctional chimeras, targeting strategies, and drug carrier approaches.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.