13 documents found, page 1 of 2

Brain-targeted drug delivery - nanovesicles directed to specific brain cells by...

Neurodegenerative diseases are characterized by extensive loss of function or death of brain cells, hampering the life quality of patients. Brain-targeted drug delivery is challenging, with a low success rate this far. Therefore, the application of targeting ligands in drug vehicles, such as lipid-based and polymeric nanoparticles, holds the promise to overcome the blood-brain barrier (BBB) and direct therapies...

Editorial: Advanced (gene and cell) therapies for central nervous system applic...

The stress granule protein G3BP1 alleviates spinocerebellar ataxia-associated d...

Polyglutamine diseases are a group of neurodegenerative disorders caused by an abnormal expansion of CAG repeat tracts in the codifying regions of nine, otherwise unrelated, genes. While the protein products of these genes are suggested to play diverse cellular roles, the pathogenic mutant proteins bearing an expanded polyglutamine sequence share a tendency to self-assemble, aggregate and engage in abnormal mol...

Editorial: Global excellence in gene and cell therapy: Europe

Editorial: Advanced (gene and cell) therapies for central nervous system applic...

Advanced therapies as defined by the European Medicines Agency (EMA) comprises strategies involving gene therapy, cell therapy, and tissue engineering. Overall, these strategies offer a wide range of possibilities to treat and cure diseases, including those affecting the central nervous system (CNS). In this line, Advanced (Gene and Cell) Therapies for Central Nervous System Applications Research Topic was inte...

Editorial: Global excellence in gene and cell therapy: Europe

Gene therapy changes protein expression in cells through the insertion of genes and/or gene expression-modulating tools, including gene editing. It can be used in the treatment of a variety of diseases, including genetic disorders, by reducing the levels of disease-causing proteins or by increasing the expression of missing or protective proteins. Cell therapy by the transplantation of cells or tissues aims to ...

The stress granule protein G3BP1 alleviates spinocerebellar ataxia-associated d...

Koppenol et al. show that overexpression of G3BP1 in cell models of SCA2 and SCA3 leads to a reduction in ataxin-2 and ataxin-3 aggregation. G3BP1 lentiviral delivery reduces motor deficits and neuropathology in preclinical models, suggesting that G3BP1 may be a potential therapeutic target for polyQ disorders. Polyglutamine diseases are a group of neurodegenerative disorders caused by an abnormal expansion of ...

Lysosomal Storage Disease-Associated Neuropathy: Targeting Stable Nucleic Acid ...

More than two thirds of Lysosomal Storage Diseases (LSDs) present central nervous system involvement. Nevertheless, only one of the currently approved therapies has an impact on neuropathology. Therefore, alternative approaches are under development, either addressing the underlying enzymatic defect or its downstream consequences. Also under study is the possibility to block substrate accumulation upstream, by ...

Successes and Hurdles in Stem Cells Application and Production for Brain Transp...

Brain regenerative strategies through the transplantation of stem cells hold the potential to promote functional rescue of brain lesions caused either by trauma or neurodegenerative diseases. Most of the positive modulations fostered by stem cells are fueled by bystander effects, namely increase of neurotrophic factors levels and reduction of neuroinflammation. Nevertheless, the ultimate goal of cell therapies ...

Understanding how birds rebuild fat stores during migration: insights from an e...

Mechanisms underlying fat accumulation for long-distance migration are not fully understood. This is especially relevant in the context of global change, as many migrants are dealing with changes in natural habitats and associated food sources and energy stores. The continental Black-tailed godwit Limosa limosa limosa is a long-distance migratory bird that has undergone a considerable dietary shift over the pas...