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A new study published in Science from the groups of Kevan Shokat at UCSF and Luke Gilbert at Arc Institute and UCSF reports the discovery of a cellular uptake pathway specifically important for larger drug molecules composed of linked subunits. This knowledge can be harnessed to create new drugs that, although they are large and complex in order to bind optimally to their targets, are efficiently taken up by target cells.

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Our mission at the Arc Institute is to accelerate scientific progress in understanding and tackling complex human diseases. We are excited to launch three parallel searches to build the other major arm of Arc: our curiosity-driven Laboratories.

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While great scientific progress has been made to understand the ongoing COVID-19 pandemic and to develop vaccines that protect against severe outcomes, effective drugs that reduce the likelihood or impact of infection lag behind vaccine efforts and remain a critically important part of our medical response to respiratory virus outbreaks. In order to develop such treatments, a deep molecular understanding of viral infection and interplay with human host cells is essential. A new collaborative effort led by Arc Institute cofounders Patrick Hsu and Silvana Konermann and UC Berkeley virologist Eva Harris has discovered hundreds of human genes and pathways that impact SARS-CoV-2 infection and revealed that airway mucin glycoproteins play a critical role in protecting the lungs from SARS-CoV-2 and other respiratory viruses.

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For the first of our Arc Investigator Profiles, we caught up with our newest Core Investigator, Luke Gilbert. In addition to his role at Arc, Luke is an Assistant Professor at UCSF, where he and his lab have pioneered the development of CRISPR-based epigenome editing tools toward a better understanding of the genetics and epigenetics underlying cancer. He’s particularly known for his work in developing CRISPRi and CRISPRa technology for turning desired genes on and off, and bringing these technologies to genome-wide and combinatorial screens to map the genetic interaction landscape of human cells.

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The role that advanced technologies play in scientific breakthroughs is hard to ignore - CRISPR, induced pluripotent stem cells, and single cell sequencing transformed entire fields of research. By the same token, the barriers to achieving quantum leaps in biological understanding are often technical in nature.

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Biomedical innovation in areas such as mRNA technology, gene editing, and cell therapies suggest that historic medical breakthroughs could be achieved in the years ahead. While the prevailing organizations for scientific research have yielded tremendous successes, we also believe that many important discoveries will be enabled by new research models. To accelerate scientific progress, we are launching the Arc Institute, a new scientific institution dedicated to the study of complex human diseases.

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