The LRRK2 gene has been implicated in Parkinson’s disease, but is too large to be expressed in viral vectors, a tool to modify cells. In order to study LRRK2 function, one needs a viral vector that permits expression of LRRK2. Helper dependent adenovirus (HdAd) can package large genes and so are an ideal viral vector system for LRRK2 expression.
The goal of this project is to design HdAd LRRK2 expression vectors that can be used by all Parkinson’s disease researchers.
This project aims to (i) produce HdAd LRRK2 viral vectors, (ii) carry out quality control of produced HdAd vectors, (iii) verify LRRK2 expression, (iv) test vectors in vivo and, subsequently, (v) make vectors available to the Parkinson's disease research community.
Impact on Diagnosis/Treatment of Parkinson’s Disease:
Currently, researchers in the Parkinson’s disease field are limited in their ability to study LRRK2 function due to the inability to express LRRK2 with viral vectors. Creating an HdAd viral vector that expresses LRRK2 will allow researchers to use this viral vector to help elucidate the mechanisms of LRRK2 function and design relevant therapeutics.
Next Steps for Development:
We expect to generate a battery of LRRK2 viral vectors that all Parkinson’s disease researchers can use in their projects and as a platform to develop novel therapeutics.