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Meeting on the Mesa 2021 | Tailored nucleases for primary hyperoxaluria

Devyn Smith, PhD, Arbor Biotechnologies, Cambridge, MA, discusses utilizing tailored nuclease-based CRISPR therapies to treat primary hyperoxaluria, a genetic condition that leads to the accumulation of oxalate in the liver and subsequent end-stage renal disease in children. Current treatments have a mixed efficacy and new genetic therapies aim to treat primary hyperoxaluria by multiplex gene knockout, where the nuclease targets two known protein targets. This interview took place at Meeting on the Mesa 2021.