Mentor: Dr. Ron Mandel
College of Medicine
"As someone pursuing a career in medicine, I believe a background in research is fundamental in understanding the process of developing new treatments and technologies. Research has opened my eyes to the steps needed to take a treatment from bench top to bed side."
- Gene Therapy
- Neurodegenerative Disorder
- Movement Disorders
Hobbies and Interests
Pleiotrophin for Treatment of HD: a Single rAAV Vector to Deliver Pleiotrophin under the Control of Doxycycline
The purpose of this study is to determine whether rAAV delivered Pleiotrophin (PTN) in the striatum of a full-length mouse model of Huntington's Disease (HD) (pQ140 mice) can slow or reverse the progressive pathology in this model. The doxycycline (dox) controlled version of the vector will be used because it represents a significant safety measure when and if PTN is used in patients and a better control for AAV infection. The study will be broken down into two parts. Part one will consist of 120 mice receiving a unilateral injection at 3 months. The mice's groups and purposes are shown in Chart 1. The mice will be euthanized and their brains will be removed. Samples will be shipped to a third party lab for striatal specific transcriptional analysis. Transcriptional dysregulation is likely the best outcome measure for this strain of mouse because the same transcripts are affected in humans. Other samples will remain in the Mandel Lab and will undergo analysis for striatal volume and stereological counting. Part two of the experiment will consist of 60 mice receiving the bilateral injections in Chart 2. These mice will undergo behavioral tests such as rearing tests and gait analysis. Their weights will also be tracked. At 18 months, these animals will be euthanized. Brain slices from these mice will be stained and used to quantify proteins in the region via the LiCor Oddyssey scanner and software.