8 research outputs found
Sustained AAV-mediated overexpression of CRF in the central amygdala diminishes the depressive-like state associated with nicotine withdrawal
Testing the possible negative association of type 1 diabetes and atopic disease by analysis of the interleukin 4 receptor gene
Transcriptomics Modeling of the Late-Gestation Fetal Pituitary Response to Transient Hypoxia
Gene Therapy for Misfolding Protein Diseases of the Central Nervous System
Protein aggregation as a result of misfolding is a common theme underlying neurodegenerative diseases. Accordingly, most recent studies aim to prevent protein misfolding and/or aggregation as a strategy to treat these pathologies. For instance, state-of-the-art approaches, such as silencing protein overexpression by means of RNA interference, are being tested with positive outcomes in preclinical models of animals overexpressing the corresponding protein. Therapies designed to treat central nervous system diseases should provide accurate delivery of the therapeutic agent and long-term or chronic expression by means of a nontoxic delivery vehicle. After several years of technical advances and optimization, gene therapy emerges as a promising approach able to fulfill those requirements. In this review we will summarize the latest improvements achieved in gene therapy for central nervous system diseases associated with protein misfolding (e.g., amyotrophic lateral sclerosis, Alzheimer’s, Parkinson’s, Huntington’s, and prion diseases), as well as the most recent approaches in this field to treat these pathologies. ELECTRONIC SUPPLEMENTARY MATERIAL: The online version of this article (doi:10.1007/s13311-013-0191-8) contains supplementary material, which is available to authorized users