Using CRISPR-Cas Screens to Reveal Fitness Genes and to Map Genetic Interactions

Genomic analyses are yielding a host of new information on the multiple genetic abnormalities associated with specific types of cancer. A comprehensive description of cancer-associated genetic abnormalities can improve our ability to classify tumors into clinically relevant subgroups and sometimes identify drivers. However, the functional significance of cancer-associated mutations is often difficult to discern. Forward genetic screens provide a powerful tool to identify genes or genetic networks that contribute to specific biological phenotypes or diseases and therefore hold great potential for elucidating gene function and revealing therapeutic targets for diseases. The advent of CRISPR-Cas technology has revolutionized genome editing in higher eukaryotes and enables efficient gene manipulation in mammalian cells. CRISPR guide-RNA libraries allow facile generation of a pool of genetically perturbed cells and its application in genetic screens has marked a major breakthrough for functional genomics in mammalian cells.
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OTHER ON-DEMAND WEBINARS

Building on a New Approach to Validate Gene Editing and Localize CAR Cassettes in T Cells

Safety and efficacy are key considerations in engineered cell therapies. Any technology that modifies the genome has the potential to cause genotoxicity. The accuracy of editing and the potential occurrence of unintended rearrangements should be assessed to understand the associated risks, such as cassette insertion in the vicinity of an oncogene or a tumor suppressor.
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Why are Lipid Formulations Commonly Used to Enhance Bioavailability?

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Enabling formulations becomes more and more necessary to obtain adequate exposure of APIs during (pre)-clinical research. Currently, most registered formulations to improve bioavailability are based on lipidic systems, which make use of intrinsic digestion processes within the GI tract, facilitating the absorption of APIs. Aside from a general absorption mechanism, lipidic systems can also promote lymphatic transport of poorly soluble drugs, such as hormones. In addition, many different indications, such as HIV therapy, oncology, immunosuppressant and even arising therapies related to medicinal cannabis (e.g. CBD) can benefit from lipid formulations. These formulations create multiple possibilities for oral administration because they can be manufactured as solutions, suspensions, emulsions, and self-(micro)emulsifying systems.
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Cell and Gene Therapies: From Concept to Commercialization

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The voyage of a cell or gene therapy from initial hypothesis to commercial delivery is complex and challenging. It requires a broad spectrum of scientific expertise, innovative technologies, and optimized processes to ensure these advanced therapeutics make it to market. In this GEN webinar, our panelists—two industry leaders—will discuss what it takes to develop a cell or gene therapy from concept to commercialization, alongside a conversation about innovations that are likely to shape the future of advanced therapy discovery and biomanufacturing.
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How to interpret clinical trial results like a pro

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Clinical trial data can be remarkably (and unnecessarily) difficult to read. Companies spin, short-sellers hawk, and everyone else is left swimming through the muck. But there is an art to reading scientific results — and you can learn it.
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