Florida International University Summer Research Internship
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College of Arts, Sciences, and Education
Miami, FL
Principal Investigator/Mentor: Dr. Marissa Perry, PhD
June - August 2021
+150 hrs
Research Abstract
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Using Personalized Medicine to Prevent Late-Onset Alzheimer’s Disease: Targeting High-Risk Genes using the CRISPR-Cas9 System
Alzheimer’s Disease (AD) affects more than 6 million Americans and kills more people than both breast and prostate cancer combined. AD is characterized by brain lesions, Ab plaques, neuronal death, and loss of synapses. Current treatments for AD are insufficient and do not provide a cure. Here, we searched for potential genes whose dysregulation is associated with AD that can be targeted by CRISPR/Cas9. Specifically, we proposed using personalized medicine for Late Onset AD patients (LOAD), which account for about 80% of AD patients and is highly heritable. Specific genes involved in myelination, the immune system, and lipid metabolism can have a direct impact on LOAD. Therefore, we proposed targeting specific genes to prevent LOAD development altogether. Genes that are associated with the dysregulation of myelination, the immune system, and lipid metabolism, account for the bulk of the known pathology of LOAD. For lipid metabolism, we proposed to target the genes involved in abnormal deposition of amyloid-β-peptides and accumulation of tau in neurofibrillary tangles, specifically the APOE gene. In addition, we proposed to target MYRF and BIN1 genes, which deregulate the production and cleavage of myelin. Lastly, we proposed to also target TREM2, C3, CD33, INPP5D, and MS4A genes, which are responsible for controlling inflammation and amyloid plaque density in the brain. Manipulation of the APOE, MYRF, BIN1, TREM2, C3, CD33, INPP5D, and MS4A genes has been shown to relieve Aβ plaques and neurofibrillary tangle buildup, myelination abnormalities, and lipid production irregularities. These results suggest that gene targeting may be an effective treatment for LOAD. This technique is an alternative to current medications that simply delay the disease from progressing. These findings introduced a potential solution to Alzheimer’s disease, which affects millions of individuals globally, and can pave the way for other neurodegenerative cures.
​Research Responsibilities
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During the summer of 2021, I was accepted into the very sought out Florida International University Summer Research Internship which was aimed at exposing high school students to research in various topics of interest. The program (virtual due to the pandemic) provided a daily online curriculum, including faculty seminars, case study groups, journal clubs, research teams, and professional development workshops.
The program allowed the various groups of students to choose from topics relating to biomedical engineering, genetics, neuroscience, etc. My group chose the topic of Biomedical and Health Sciences. I worked alongside 3 other students and we were all supervised by a graduate student pursuing her PhD at the time.
We were responsible for developing a hypothesis, gathering information related to the topic and analyzing a hypothetical research project aimed at solving a real world problem. Our group studied the use of CRISP-Cas9 technology as a device for personalized medicine that would work towards curing or slowing the progression of Alzheimer's Disease.
We presented our findings at the cohort and received the Audience Choice Award. We also submitted out research paper to the Florida Science and Engineering Fair where it won a first place and "Superior" ranking award.
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Using Personalized Medicine to Prevent Late-Onset Alzheimer’s Disease: Targeting High-Risk Genes using the CRISPR-Cas9 System