Going beyond CRISPR with 1 million edits per cell │ Dr. George Church
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CRISPR-Cas9 is a system of gene editing that has revolutionized how scientists engineer cells over the past decade. Before CRISPR, homologous recombination and single-stranded DNA annealing proteins (SSAPs) enabled early gene editing, but what comes next? Deaminases, transposonases, and more sophisticated SSAPs are able to deliver gene therapies with more precision than CRISPR.
Multiplex editing allows geneticists to make multiple genome alterations at once. Using this amazing technology, humanized organs from large mammals like pigs will enable organ transplantation on demand instead of patients waiting for years for a donor. Base editing in humans may facilitate gene therapies that eliminate common diseases of aging such as high cholesterol. Unlike antibody treatments that target the PCSK9 protein (an important regulator of blood cholesterol levels), which are very expensive, base editing could deliver permanent protection from hypercholesterolemia with just one dose. In this clip, Dr. George Church shares insights on the future of gene therapies in healthcare.
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