The Science Against SARS-CoV-2 event kicked off with presentations from key thought leaders from prestigious universities and institutions from around the globe.
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COVID-19 represents a global crisis, yet significant knowledge gaps remain about human immunity to SARS-CoV-2. As many researchers raced to develop the vaccines and therapeutics needed to mitigate the pandemic, other investigators took up the task of understanding the human body’s diverse response to this virus. With technology advancing exponentially, researchers have an arsenal of analytic tools now at their disposal to decipher the most elusive aspects of viral immunobiology.
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Genome-editing enzymes such as CRISPR-Cas9 have been successfully employed to target different genetic disorders in model systems, and ex vivo approaches have demonstrated the clinical potential of this technology. However, the challenge of delivering such corrective enzymes in vivo represents a substantial barrier to therapeutic translation. Viral vectors—refined during their use in traditional gene therapy—represent the most widely-used delivery platform in preclinical work, and nanoparticles are quickly maturing into a comparably powerful vehicle. Direct delivery of pre-formed CRISPR enzymes has recently emerged as an appealing strategy for enabling therapeutic editing in vivo. In this GEN webinar, we will hear how the Wilson lab works to advance this approach by performing molecular engineering, giving CRISPR enzymes the properties necessary for safe, efficient, and accessible delivery in a clinical setting. These efforts will enable in vivo delivery of CRISPR enzymes, including hard-to-deliver tools such as base editors and prime editors.
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Antibodies have established themselves as one of the most important classes within the biologics space, and developing these therapeutics requires a series of successful decisions throughout the early-discovery process.
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