Purpose and goal
The aim of our project is to develop innovative treatments for pyruvate dehydrogenase complex (PDC) deficiency, a serious metabolic disorder that affects approximately 1 in 40,000 children and often results in early mortality. PDC deficiency is often caused by genetic mutations that impair cellular energy production. Current treatment options are limited and provide inconsistent results, underscoring the urgency of developing new, targeted therapies.
Expected effects and result
This project will benefit from the expertise of both OligoNova and PTZL AB to develop a therapeutic siRNA. OligoNova is responsible for conducting the in silico prediction, design and synthesis of a library of siRNAs, which will then be tested in vitro to assess their gene silencing efficiency and specificity. PTZL AB will contribute valuable insights into the therapeutic target and contribute biological expertise to keep the project in line with its objective.
Planned approach and implementation
In the first phase, OligoNova will develop small interfering RNAs (siRNAs) designed to target a mitochondria-related gene identified by PTZL AB as a potential treatment target for pyruvate dehydrogenase complex (PDC) deficiency.
In the second phase, OligoNova will refine the siRNA to maximize potency and reduce off-target effects. PTZL AB will contribute specialized functional analyzes and conduct preliminary in vivo tests.
The project description has been provided by the project members themselves and the text has not been looked at by our editors.
