Publications by authors named "Paul D Ross"
Article Synopsis
- Delivering large therapeutic proteins effectively across biological barriers like the blood-brain barrier is challenging, but Toxoplasma gondii, a parasite, shows promise in overcoming this issue by naturally moving from the gut to the central nervous system.
- Researchers have engineered T. gondii's secretion systems to facilitate the delivery of these proteins into neurons, testing their effectiveness in lab cultures, brain organoids, and living mice.
- The study specifically highlights the successful delivery of the MeCP2 protein, which could be a potential treatment for Rett syndrome, while also discussing the system's limitations and potential for future improvements.
Article Synopsis
- - Rett syndrome (RTT) is a serious genetic disorder caused by mutations in the MECP2 gene, primarily affecting the nervous system and causing various neurological issues.
- - Researchers created mice with normal MECP2 levels in the nervous system but low levels in other tissues to explore these non-neuronal contributions to RTT symptoms.
- - Findings showed that most behavioral and physiological RTT symptoms were linked to the brain, but some peripheral issues, like hypoactivity and bone problems, suggest that RTT also has effects outside the nervous system.
Rett syndrome (RTT) is a genetic disorder characterized by a range of features including cognitive impairment, gait abnormalities and a reduction in purposeful hand skills. Mice harbouring knockout mutations in the Mecp2 gene display many RTT-like characteristics and are central to efforts to find novel therapies for the disorder. As hand stereotypies and gait abnormalities constitute major diagnostic criteria in RTT, it is clear that motor and gait-related phenotypes will be of importance in assessing preclinical therapeutic outcomes.
View Article and Find Full Text PDFTypical Rett syndrome (RTT) is a pediatric disorder caused by loss-of-function mutations in the methyl-CpG binding protein 2 (MECP2) gene. The demonstrated reversibility of RTT-like phenotypes in mice suggests that MECP2 gene replacement is a potential therapeutic option in patients. We report improvements in survival and phenotypic severity in Mecp2-null male mice after neonatal intracranial delivery of a single-stranded (ss) AAV9/chicken β-actin (CBA)-MECP2 vector.
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