Research Area #5: The Golde ”lab moonshot”
The current standard paradigm for development of disease modifying therapies for neurodegenerative diseases is to develop a single therapeutic agent, often targeting the presumptive trigger of the disease and test that agent in symptomatic patients. To date, the success of this approach has been very underwhelming. In order to dramatically increase the likelihood of success and decrease the time needed to develop therapies that offer substantial clinical benefit to symptomatic patients suffering from Alzheimer’s Disease and related disorders (ADRDs) or other incurable disorders, we are evaluating novel paradigms that account for and effectively target the long-standing and severe damage that underlies the symptomatic phase of disease. Our approach, which we refer to as Precision AAV-mediated Combinatorial Therapy for ADRDs untreatable diseases, is designed to build a much-needed road map for development of combinatorial therapies needed to appreciably alter the clinical trajectory in the face of long-standing incurable disease. This new imitative leverages our rAAV toolkit that enables us to both design and deliver multiple biotherapeutics at the same time.
Funding NIH U01AG046139
