Blue Sky Proposal Paper

Words: 443
Pages: 2

Blue Sky Proposal: Explore using CRISPR gene editing as a solution to combat the HIV virus.

HIV is a retrovirus that primarily infects the immune system. Once inside the cell, HIV converts the viral RNA into DNA. This DNA is then transported into the cell's nucleus where it is inserted into the human DNA. HIV has caused an estimated 36 million deaths worldwide and it is estimated that another 35 million people are currently living with the disease. I aim to explore using CRISPR gene editing as a solution to combat the virus. CRISPR gene editing allows us to replace targeted gene sequences with new DNA sequences that I believe could be a potential strategy to combat HIV.

A major hurdle with this approach is the fact that it is still not clear on how to find the virus in latently infected cells. Researchers at the UMass Medical School are currently working on using a combination of innovative technologies to describe and model HIV DNA integrated into the genome of reservoir cells. Characterizing the genomic landscape of these latently infected cells will allow the researchers to identify vulnerable and accessible genetic sequences that can be potentially cut out of the HIV virus to make it permanently inactive.
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Regarding the excising of the gene itself I would aim to use 2 RNAs to find and cleave both LTR (3’ and the 5’) ends of the virus gene. This according to Yoshio Koyangi of Kyoto University would be a lot more efficient as compared to a single gRNA expression strategy with Cas9 nucleus that he has been carrying out research on. With any kind of CRISPR another challenge is the delivery, and a method to find the specific infected cells has to be devised. To explore this I will examine Kamel Khalili from the University of Pennsylvania’s research in developing a nanoparticle delivery