2018 Research Updates
Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents. This study focuses on identifying a miRNA that directly downregulates expression of the mutant AR transgene and on designing a safe and efficient mode of delivery of the miRNA to the spinal cord and skeletal muscle of our disease mouse model. For onformation on this study, click here.
Carlo Rinaldi, associate professor and clinician scientist at the University of Oxford in England, was awarded an MDA Development Grant totaling $120,000 over 3 years to study the role of androgen receptor isoforms in SBMA. The MDA just announced it is funding 34 new research projects totaling 9.9 million dollars. For more information, click here.
Study identifies potential drug for treatment of Kennedy's Disease. To read the Medical Press article, click here