Prof. Manuela Basso's working group at CIBIO has won funding under the Telethon Foundation's 'multi-round' call for proposals to study Kennedy's disease.
This disease is caused by a mutation in the androgen receptor (AR). The AR is the receptor for the male hormone testosterone, and only males show symptoms of the disease. The AR acts as a transcription factor, i.e. it controls the function of other genes, playing a key role in maintaining cell health.
This project aims to demonstrate that, by acting on these co-activators, it is possible to reduce the malfunctioning of the AR receptor while preserving its normal physiological function, which is necessary for cell survival. To dampen the altered activity of the two co-activators, adeno-associated viruses, and nanoparticles capable of delivering therapeutic molecules into the diseased muscle will be used.
Through this project, knowledge is being advanced toward developing new therapeutic strategies for Kennedy's disease.