Periodic Reporting for period 1 - FAST-DEVELOPS (Developing new therapeutics for Friedreich ataxia)
Okres sprawozdawczy: 2015-11-01 do 2017-04-30
The FAST-DEVELOPS project has allowed the establishment of an experimental systems that is instrumental for the validation of potential new drugs for Friedreich ataxia.
As set as the goal of this project, in fact, we developed the cell culture conditions that allow the differentiation of inducible pluripotent stem cells derived from Friedreich ataxia patients into sensory neurons. The successful establishment of this system enables the validation of compounds with potential therapeutic efficacy in cells that are relevant for the disease. In fact we can now measure the ability of the new compounds we are developing to elevate frataxin levels in Friedreich ataxia patient's derived sensory neurons and measure their ability to restore key metabolic pathways that are impaired in these cells. This system can be used for the validation of any potential therapeutic for Friedreich ataxia and is considered crucial for the progression of drug discovery programs into clinical phases.
As set as the goal of this project, in fact, we developed the cell culture conditions that allow the differentiation of inducible pluripotent stem cells derived from Friedreich ataxia patients into sensory neurons. The successful establishment of this system enables the validation of compounds with potential therapeutic efficacy in cells that are relevant for the disease. In fact we can now measure the ability of the new compounds we are developing to elevate frataxin levels in Friedreich ataxia patient's derived sensory neurons and measure their ability to restore key metabolic pathways that are impaired in these cells. This system can be used for the validation of any potential therapeutic for Friedreich ataxia and is considered crucial for the progression of drug discovery programs into clinical phases.