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“So, given our demonstration that this form is not as toxic as previously believed, a completely different treatment strategy is required if followed by removal of alternative or mutated forms.” she explained.
Sheikh juggles a variety of roles at UC San Diego, balancing time between research, teaching, and serving on various committees, while supporting the lab’s diverse team of trainees, an academic team of project scientists and postdocs. I teach a wide range of groups from all over the world. From researchers to graduate students and even undergraduate students. Reflecting on the dynamic nature of her responsibilities, if she were to draw a pie chart of her life at UC San Diego as a researcher, educator, mentor, entrepreneur, collaborator, and consultant, each day would be a different pie. Deaf, she says.
“Most of my efforts are in the research area, working with amazing trainees and seeing their joy in the results and in moving the needle forward,” she said.
When Sheikh helped ring the bell for LEXEO on Nasdaq, she was also helping move the needle forward and usher in a new era for cardiac gene therapy innovation. “I think we’re at a pivotal moment in time where gene therapy can be used safely in patients,” she says.
Sheikh’s ARVC approach is based on engineering a non-replicating virus that targets the PKP2 gene. Although many labs targeting other genetic diseases use similar approaches, her strategy is uniquely created to address the complexities of ARVC. She notes that her concerns from her previous studies about safety and side effects such as liver toxicity were largely resolved by simply reducing the dose.
“I think we are at a great time to take advantage of this technology,” Sheikh said. “For genetic diseases where a single gene may be responsible for a particular genetic disease, we have all the data to show that we can start addressing them at this stage.”
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