Publications by authors named "P Santonicola"
Purpose: Dimethyl fumarate (DMF), the first-line oral therapy for relapsing-remitting multiple sclerosis, is rapidly metabolized into monomethyl fumarate. The DMF oral administration provokes gastrointestinal discomfort causing treatment withdrawal. The present study aimed to develop an innovative formulation for DMF nasal administration.
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- * The study used advanced techniques such as proteomics and interactomics on mouse models with varying disease severities to identify networks of proteins and their interactions that illuminate both similarities and differences in SMA presentation.
- * By integrating data on the SMN protein with these molecular networks, researchers were able to pinpoint critical connections and disruptions that contribute to SMA, offering a new way to analyze monogenic diseases through a systematic approach.
Extracellular vesicles (EVs) are membrane-enclosed bio-nanoparticles secreted by cells and naturally evolved to transport various bioactive molecules between cells and even organisms. These cellular objects are considered one of the most promising bio-nanovehicles for the delivery of native and exogenous molecular cargo. However, many challenges with state-of-the-art EV-based candidates as drug carriers still exist, including issues with scalability, batch-to-batch reproducibility, and cost-sustainability of the final therapeutic formulation.
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- Wilson disease (WD) is caused by mutations in the ATP7B gene, leading to copper overload in the liver and brain, which can result in severe health issues.
- A mutant strain of Caenorhabditis elegans was created to study this condition, showing significant Cu sensitivity, stunted development, and other health impairments due to a specific ATP7B variant.
- The cua-1 mutant strain serves as a valuable experimental model for understanding copper toxicity in WD and testing potential therapeutic approaches.
According to the amyloid hypothesis, in the early phases of Alzheimer's disease (AD), small soluble prefibrillar aggregates of the amyloid β-peptide (Aβ) interact with neuronal membranes, causing neural impairment. Such highly reactive and toxic species form spontaneously and transiently in the amyloid building pathway. A therapeutic strategy consists of the recruitment of these intermediates, thus preventing aberrant interaction with membrane components (lipids and receptors), which in turn may trigger a cascade of cellular disequilibria.
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