Regulated gene expression for Huntington's disease therapy

The aim of the project is to develop the gene therapy against Huntington's Disease by

• preventing the production of the mutated Huntingtin protein with RNA interference (RNAi)
• stimulate the maintenance and survival of neurons by neurotrophic factors like glial cell line-derived neurotrophic factor (GDNF)

 The global molecular state of the cells that have been challenged with the viral vectors delivering the intended therapeutic nucleic acid will be monitored by means of total gene expression analysis (SAGE) and monitoring of small RNA landscape, by high-throughput DNA sequencing technology (NGS- next generation sequencing)