BREATHE Members Publish New Study on Neonatal Systemic Gene Therapy and its Role in Restoring Cardiorespiratory Function in a Rat Model of Pompe Disease
A new study, led by Dr. David Fuller, shows restoration of cardio-respiratory function following neonatal AAV9 gene therapy in a rat model of Pompe disease. Since central nervous system disease substrates are missed using enzyme replacement therapy, this approach mitigates critical disease causing neuropathology. Check out the full publication…