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Non-Viral Systems Based on PAMAM-Calix-Dendrimers for Regulatory siRNA Delivery into Cancer Cells.
Int J Mol Sci
November 2024
A.M. Butlerov Chemistry Institute, Kazan Federal University, 18 Kremlyovskaya Str., 420008 Kazan, Russia.
Cancer is one of the most common diseases in developed countries. Recently, gene therapy has emerged as a promising approach to cancer treatment and has already entered clinical practice worldwide. RNA interference-based therapy is a promising method for cancer treatment.
View Article and Find Full Text PDFCross-species RNAi therapy via AAV delivery alleviates neuropathic pain by targeting GCH1.
Neurotherapeutics
December 2024
Department of Neurosurgery, Yonsei University College of Medicine, Seoul, Republic of Korea; Department of Neurosurgery, Brain Research institute, Yonsei University College of Medicine, Seoul, Republic of Korea. Electronic address:
Tetrahydrobiopterin (BH4) expression is normally strictly controlled; however, its intracellular levels increase considerably following nerve damage. GTP cyclohydrolase I (GCH1) plays a crucial role in regulating BH4 concentration, with an upregulation observed in the dorsal root ganglion in cases of neuropathic pain. In this study, we aimed to develop and evaluate the clinical potential of an RNA interference-based adeno-associated virus (AAV) targeting GCH1 across various species to decrease BH4 levels and, consequently, alleviate neuropathic pain symptoms.
View Article and Find Full Text PDFDiving into RNAi Therapy: An Inhalable Formulation Based on Lipid-Polymer Hybrid Systems for Pulmonary Delivery of siRNA.
Biomacromolecules
January 2025
Lab of Biocompatible Polymers, Department of Biological, Chemical and Pharmaceutical Sciences and Technologies (STEBICEF), University of Palermo, Via Archirafi 32, Palermo 90123, Italy.
Here, a pulmonary formulation based on lipid-polymer hybrid nanoparticles carrying small interfering RNA (siRNA) was developed to realize a RNA interference-based therapy to treat respiratory diseases. Toward this aim, a new copolymer was synthesized, by functionalization of the α,β-poly(-2-hydroxyethyl)-d,l-aspartamide with 35 mol % of 1,2-bis(3-aminopropylamino)ethane, 0.4 mol % of fluorescent dye, and 4.
View Article and Find Full Text PDFAltered Ca2+ responses and antioxidant properties in Friedreich's ataxia-like cerebellar astrocytes.
J Cell Sci
January 2025
Division of Neuroscience, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.
Friedreich's ataxia (FRDA) is a neurodegenerative disorder characterized by severe neurological signs, affecting the peripheral and central nervous system, caused by reduced frataxin protein (FXN) levels. Although several studies have highlighted cellular dysfunctions in neurons, there is limited information on the effects of FXN depletion in astrocytes and on the potential non-cell autonomous mechanisms affecting neurons in FRDA. In this study, we generated a model of FRDA cerebellar astrocytes to unveil phenotypic alterations that might contribute to cerebellar atrophy.
View Article and Find Full Text PDFPotential pesticide substrates of an insect ABCC transporter.
Insect Sci
November 2024
Shenzhen Branch, Guangdong Laboratory of Lingnan Modern Agriculture, Genome Analysis Laboratory of the Ministry of Agriculture and Rural Affairs, Agricultural Genomics Institute at Shenzhen, Chinese Academy of Agricultural Sciences, Shenzhen, China.
Article Synopsis
- The use of synthetic pesticides can lead to pests developing resistance, making these pesticides less effective over time, largely due to efflux pumps like the ABCC transporters.
- In this study, researchers identified the potential pesticide substrates of a specific transporter, TcABCC-9C, from the red flour beetle and found that the insecticides carbofuran and buprofezin bind to it.
- The results highlight TcABCC-9C's important role in developing multi-pesticide resistance, suggesting it could be a target for future pest management strategies, and the methodology could help in understanding other transporter-related insecticide resistances.
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