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Research Specialist
We are forming a team in the Harvard Medical School Department of Neurobiology dedicated to repurposing natural mechanisms of gene regulation to engineer precision viral vectors for gene therapy. First-generation gene therapy vectors lack optimal regulatory elements that lead to sub-therapeutic levels of expression and off-target toxicity. Our goal is to employ our in-house single-cell screening platform (PESCA, Hrvatin, et al., 2019), to sift through the mammalian genome and identify regulatory elements capable of driving optimal patterns of viral transgene expression to address a wide range of diseases. We are building a technology platform that fills a critical void in the gene therapy space. Our collective mission is to employ these regulatory elements to create novel
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