• Filter By:
• Specialty
• Source
• Condition
• Cancer
• Infectious Disease
• Drug
• Training
• Management
• Procedure
• Therapy
• Vaccine
• Nutrition
• Country

Modified cyclodextrin-based nanoparticles mediated delivery of siRNA for Huntingtin gene silencing across an in vitro BBB model
In conclusion, the CD platform is a promising option for delivery of siRNA-based therapeutics for HD with wider potential to treat other diseases with a genetically validated target in the central nervous system.PMID:34793942 | DOI:10.1016/j.ejpb.2021.11.003
Source: European Journal of Pharmaceutics and Biopharmaceutics - November 18, 2021 Category: Drugs & Pharmacology Authors: Monique C P Mendon ça Michael F Cronin John F Cryan Caitriona M O'Driscoll Source Type: research

Current Development of siRNA Bioconjugates: From Research to the Clinic
In this study, it was shown that the main factor determining the nature of the biodistribution of conjugates is their lipophilicity. Conjugates of siRNA with lower lipophilicity; i.e., derivatives of retinoic acid, lithocholic acid, and docosahexanoic acid with greater efficiency than cholesterol conjugates accumulated in the kidneys, bladder, and lungs of the mouse after subcutaneous injection (Biscans et al., 2018). This fact is consistent with previous data that showed that more lipophilic conjugates bind more efficiently to serum components, and thus are not excreted by the kidneys (Wolfrum et al., 2007; Osborn et al.,...
Source: Frontiers in Pharmacology - April 25, 2019 Category: Drugs & Pharmacology Source Type: research

Neurodegenerative disorders: New Huntington disease target identified
Nature Reviews Drug Discovery 14, 386 (2015). doi:10.1038/nrd4652 Author: Sarah Crunkhorn Huntington disease (HD) is caused by the expansion of a CAG repeat tract in the gene encoding huntingtin (HTT), resulting in aggregation of a toxic mutant HTT protein (mHTT). Using an siRNA-based screen in human cultured cells, followed by hit validation in Drosophila melanogaster,
Source: Nature Reviews Drug Discovery - May 15, 2015 Category: Drugs & Pharmacology Authors: Sarah Crunkhorn Tags: Research Highlight Source Type: research

Current Therapeutic Advances in Patients and Experimental Models of Huntington's Disease.
Abstract Huntington's disease (HD) clinical manifestations begin insidiously and are progressively incapacitating. Symptomatic therapies, in particular dopamine blockers and neuroleptics, are presently the only treatment for HD. Identification of neuropathological mechanisms that underlie the selective striatal and cortical neurodegeneration has allowed for the development of novel neuroprotective therapies that may improve HD patients' quality of life and enhance their survival. In this review we describe the symptomatic and neuroprotective therapies in HD that are currently in a pre-clinical or clinical stage. N...
Source: Current Drug Targets - November 24, 2013 Category: Drugs & Pharmacology Authors: Brett AC, Rosenstock TR, Rego AC Tags: Curr Drug Targets Source Type: research