The endosomal escape vehicle platform enhances delivery of oligonucleotides in preclinical models of neuromuscular disorders [PDF]
Molecular Therapy: Nucleic Acids, 2023 Biological therapeutic agents are highly targeted and potent but limited in their ability to reach intracellular targets. These limitations often necessitate high therapeutic doses and can be associated with less-than-optimal therapeutic activity.
Xiang Li +15 more
doaj +4 more sources
HOPS-Dependent Endosomal Escape Demands Protein Unfolding [PDF]
ACS Central ScienceThe inefficient translocation of proteins across biological membranes limits their application as potential therapeutics and research tools. In many cases, the translocation of a protein involves two discrete steps: uptake into the endocytic pathway and endosomal escape.
Madeline Zoltek +5 more
doaj +7 more sources
Limiting endosomal damage sensing reduces inflammation triggered by lipid nanoparticle endosomal escape. [PDF]
Nat NanotechnolOmo-Lamai S +38 more
europepmc +5 more sources
Modular Calcium‐Responsive and CD9‐Targeted Phospholipase System Enhancing Endosomal Escape for DNA Delivery [PDF]
Advanced ScienceGene delivery systems must overcome multiple barriers, with endosomal escape representing a prominent obstacle. We have previously shown that a bacterial phospholipase C (PLC) enabled endosomal escape of a non‐viral protein‐based DNA delivery system ...
Alexander Klipp +5 more
doaj +3 more sources
Unravelling cytosolic delivery of endosomal escape peptides with a quantitative endosomal escape assay (SLEEQ) [PDF]
ACS Applied Materials & Interfaces, 2020 AbstractEndosomal escape is an essential requirement but a major obstacle to efficient delivery of therapeutic peptides, proteins and nucleic acids. Current understanding of endosomal escape mechanisms remains limited due to significant number of conflicting reports, which are compounded by low sensitivity and indirect assays.
Serena L. Y. Teo +5 more
+5 more sources
Endosomal escape of delivered mRNA from endosomal recycling tubules visualized at the nanoscale [PDF]
Journal of Cell Biology, 2021 AbstractDelivery of exogenous mRNA using lipid nanoparticles (LNP) is a promising strategy for therapeutics. However, a bottleneck remains the poor understanding of the parameters that correlate with endosomal escape vs. cytotoxicity. To address this problem, we compared the endosomal distribution of six LNP-mRNA formulations of diverse chemical ...
Prasath Paramasivam +13 more
openalex +6 more sources
Stochastic Model of Virus–Endosome Fusion and Endosomal Escape of pH-Responsive Nanoparticles [PDF]
Mathematics, 2022 In this paper, we set up a stochastic model for the dynamics of active Rab5 and Rab7 proteins on the surface of endosomes and the acidification process that govern the virus–endosome fusion and endosomal escape of pH-responsive nanoparticles. We employ a
Sergei Fedotov +3 more
doaj +4 more sources
pH-Responsive Amphiphilic Carboxylate Polymers: Design and Potential for Endosomal Escape [PDF]
Frontiers in Chemistry, 2021 The intracellular delivery of emerging biomacromolecular therapeutics, such as genes, peptides, and proteins, remains a great challenge. Unlike small hydrophobic drugs, these biotherapeutics are impermeable to the cell membrane, thus relying on the ...
Shiqi Wang
doaj +4 more sources
Imaging small molecule-induced endosomal escape of siRNA [PDF]
Nature Communications, 2020 Therapeutic siRNA becomes trapped in endosomes, limiting its efficacy. Here the authors use fluorescently-tagged galectin-9 as a biosensor for membrane damage to monitor endosomal escape of cholesterol-conjugated siRNA following treatment of small ...
Hampus Du Rietz +4 more
doaj +3 more sources
Nanoscopy for endosomal escape quantification. [PDF]
Nanoscale Adv, 2021 How nanoscopy can be applied towards the study and quantification of endosomal escape of nanoparticles.
Andrian T +3 more
europepmc +5 more sources