Application of siRNA to knockdown a specific gene requires target mRNA accessibility, effective intracellular delivery of siRNA into target cells and potent siRNA inhibition of target mRNA. Use of siRNA as a tool is advancing in almost every field of biomedical research, but some of the most dynamic and exciting applications of siRNA are in cancer research. This review summarizes the results obtained with siRNA in cancer, in particular functional validation of tumorigenic genes in cell culture and animal tumor models, effective siRNA delivery systems, efficiency of siRNA agents compared with antisense oligonucleotides and efforts for potential therapeutic development. Along with the rapidly growing literature on using siRNA as a functional genomic tool, there is emerging evidence that siRNA may represent a novel therapeutic modality for cancer treatment when optimized local and systemic delivery systems are available.
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Alzheimers Dement
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Indiana University School of Medicine, Indianapolis, IN, USA.
Background: The TaRget Enablement to Accelerate Therapy Development of Alzheimer's Disease (TREAT-AD) Centers are dedicated to identifying and validating targets from the NIH Accelerating Medicines Partnership for Alzheimer's Disease (AMP-AD). The centers develop Target Enabling Packages (TEPs) to explore new therapeutic target hypotheses, moving beyond the traditional focus on amyloid or tau pathologies. In accordance with open science principles, data, methods, and tools are freely shared with the research community via an open-access platform, the AD Knowledge Portal.
View Article and Find Full Text PDFLight-activated nanocomposite thin sheet for high throughput contactless biomolecular delivery into hard-to-transfect cells.
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January 2025
Department of Engineering Design, Indian Institute of Technology Madras, India.
High throughput intracellular delivery of biological macromolecules is crucial for cell engineering, gene expression, therapeutics, diagnostics, and clinical studies; however, most existing techniques are either contact-based or have throughput limitations. Herein, we report a light-activated, contactless, high throughput photoporation method for highly efficient and viable cell transfection of more than a million cells within a minute. We fabricated reduced graphene oxide (rGO) nanoflakes that was mixed with a polydimethylsiloxane (PDMS) nanocomposite thin sheet with an area of 3 cm and a thickness of ∼600 μm.
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Int J Med Sci
January 2025
Department of Cardiology, The Second Xiangya Hospital, Central South University, Changsha 410011, Hunan Province, People's Republic of China.
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View Article and Find Full Text PDFBMSCs-derived small extracellular vesicles antagonize cerebral endothelial Caveolin-1 driven autophagic degradation of tight-junction proteins to protect blood-brain barrier post-stroke.
Int J Biol Sci
January 2025
Institute of Chinese Medical Sciences, State Key Laboratory of Quality Research in Chinese Medicine, University of Macau, Taipa, Macau SAR, China.
Bone marrow mesenchymal stem cells (BMSCs) -derived extracellular vesicles (EVs), especially small EVs (sEVs), were vastly reported to enable multiple restorative effects on ischemic stroke, yet the protective mechanism of blood-brain barrier (BBB) has not been fully illustrated. In the present study, we investigated the therapeutic effects and mechanism of BMSCs-derived sEVs on BBB injury after ischemic stroke. In-vivo, administering sEVs to transient middle cerebral artery occlusion (tMCAo) mice mitigated the brain infarct volume, BBB permeability and neural apoptosis, and improved the cerebral blood flow perfusion and neurological function.
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