ReviewAdvanced drug delivery reviews2023
Polymer-based drug delivery systems under investigation for enzyme replacement and other therapies of lysosomal storage disorders.
Review in Advanced drug delivery reviews, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 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.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
11 citing papers in PubMed, 25 citations in OpenAlex.
- Recent Advances in Diversified Materials for Spinal Cord Injury Repair and Regeneration.Gels (Basel, Switzerland) · 2026Review
- Polymeric lysosome-targeting chimeras for extracellular α-synuclein degradation in Parkinson's disease.Acta neuropathologica communications · 2026Review
- Oxidative Stress and Lysosomal Dysfunction in Neurodegenerative Diseases: Underlying Mechanisms and Nanotherapeutic Targeting Strategies.Antioxidants (Basel, Switzerland) · 2026Review
- Engineering B cells to treat and study human disease.Nature biotechnology · 2025Review
- Paclitaxel-Loaded Polyelectrolyte Nanocarriers: Uptake Mechanisms, Cytotoxicity, and Genotoxicity in Human Endothelial and Breast Cancer Cells.Nanotechnology, science and applications · 2025Article
- New Cellular Models to Support Preclinical Studies on ICAM-1-Targeted Drug Delivery.Journal of drug delivery science and technology · 2024Article
- Peptide-coated DNA nanostructures as a platform for control of lysosomal function in cells.Chemical engineering journal (Lausanne, Switzerland : 1996) · 2024Article
- Impact of mechanical cues on key cell functions and cell-nanoparticle interactions.Discover nano · 2024Review
- Novel Fused Pyrazole Amide Analogs as Glucosylceramide Synthase Inhibitors for Treating Lysosomal Storage Diseases, Neurodegenerative Diseases, Cystic Diseases, and Cancer.ACS medicinal chemistry letters · 2023Article
- Role of the Lactide:Glycolide Ratio in PLGA Nanoparticle Stability and Release under Lysosomal Conditions for Enzyme Replacement Therapy of Lysosomal Storage Disorders.Journal of functional biomaterials · 2023Article
- Fused Pyrazole Urea Analogs as Glucosylceramide Synthase Inhibitors for Treating Diseases.ACS medicinal chemistry letters · 2023Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors at 2 institutions in 2 countries.
Funding
Abstract
Lysosomes play a central role in cellular homeostasis and alterations in this compartment associate with many diseases. The most studied example is that of lysosomal storage disorders (LSDs), a group of 60 + maladies due to genetic mutations affecting lysosomal components, mostly enzymes. This leads to aberrant intracellular storage of macromolecules, altering normal cell function and causing multiorgan syndromes, often fatal within the first years of life. Several treatment modalities are available for a dozen LSDs, mostly consisting of enzyme replacement therapy (ERT) strategies. Yet, poor biodistribution to main targets such as the central nervous system, musculoskeletal tissue, and others, as well as generation of blocking antibodies and adverse effects hinder effective LSD treatment. Drug delivery systems are being studied to surmount these obstacles, including polymeric constructs and nanoparticles that constitute the focus of this article. We provide an overview of the formulations being tested, the diseases they aim to treat, and the results observed from respective in vitro and in vivo studies. We also discuss the advantages and disadvantages of these strategies, the remaining gaps of knowledge regarding their performance, and important items to consider for their clinical translation. Overall, polymeric nanoconstructs hold considerable promise to advance treatment for LSDs.
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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.