Immune-modulatory biomimetic nanoparticles: Advances in design, mechanisms, and nanomedicine applications [PDF]
BioImpactsThe development of biomimetic nanoparticles (NPs) represents an innovative approach to address the lacks of conventional drug delivery systems. This advancement demonstrates promising potential in immunotherapy.
Somayeh Vandghanooni +2 more
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Induction of WNT16 via peptide-mRNA nanoparticle-based delivery maintains cartilage homeostasis [PDF]
, 2020 Osteoarthritis (OA) is a progressive joint disease that causes significant disability and pain and for which there are limited treatment options. We posit that delivery of anabolic factors that protect and maintain cartilage homeostasis will halt or ...
Akk, Antonina +6 more
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Genome editing in the adrenal gland: a novel strategy for treating congenital adrenal hyperplasia [PDF]
Exploration of Endocrine and Metabolic DiseasesCongenital adrenal hyperplasia due to 21-hydroxylase deficiency leads to high morbidity and mortality, despite the availability of life-saving corticosteroid replacement therapy.
Eva B. van Dijk +3 more
doaj +1 more source
Toward the full potential of mRNA therapeutics. [PDF]
Mol TherMcNamara JO, Giangrande PH.
europepmc +3 more sources
Insights into the kinetics of siRNA-mediated gene silencing from live-cell and live-animal bioluminescent imaging [PDF]
, 2006 Small interfering RNA (siRNA) molecules are potent effectors of post-transcriptional gene silencing. Using noninvasive bioluminescent imaging and a mathematical model of siRNA delivery and function, the effects of target-specific and treatment-specific ...
Bartlett, Derek W., Davis, Mark E.
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A novel protein isoform of the RON tyrosine kinase receptor transforms human pancreatic duct epithelial cells. [PDF]
, 2016 The MST1R gene is overexpressed in pancreatic cancer producing elevated levels of the RON tyrosine kinase receptor protein. While mutations in MST1R are rare, alternative splice variants have been previously reported in epithelial cancers.
Babicky, M +10 more
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mRNA Therapeutic Modalities Design, Formulation and Manufacturing under Pharma 4.0 Principles
Biomedicines, 2021 In the quest for a formidable weapon against the SARS-CoV-2 pandemic, mRNA therapeutics have stolen the spotlight. mRNA vaccines are a prime example of the benefits of mRNA approaches towards a broad array of clinical entities and druggable targets ...
Andreas Ouranidis +7 more
doaj +1 more source
Tailoring Design of Nanomaterials and Systems to Individualize Patient Treatments
CHIMIA, 2022 Nanomedicine encompasses usage of materials smaller than 100 nm for diagnosis, monitoring and treatment of disease. A frequent application of these materials is in reformulation of active drugs, which were previously approved for clinical use.
Marija Buljan, Peter Wick
doaj +1 more source
Modulation of the GABAergic pathway for the treatment of fragile X syndrome. [PDF]
, 2014 Fragile X syndrome (FXS) is the most common genetic cause of intellectual disability and the most common single-gene cause of autism. It is caused by mutations on the fragile X mental retardation gene (FMR1) and lack of fragile X mental retardation ...
Hagerman, Randi J +2 more
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Racing against COVID-19: a vaccines strategy for Europe. Bruegel Policy Contribution Issue n˚7 | April 2020 [PDF]
, 2020 The fast development of vaccines is an essential part of the long-term solution to COVID-19, but vaccine development has high costs and carries the risk of high failure rates.
Veugelers, Reinhilde, Zachmann, Georg.
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