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Explore cutting-edge research presentations from the Ladders to Cures Accelerator Symposium focused on translating genetic discoveries into clinical treatments for rare diseases. Learn about breakthrough developments in gene editing and genetic insights through two featured presentations: discover how in vivo prime editing technology successfully rescues alternating hemiplegia of childhood in mouse models, and examine genetic insights combined with Perturb-seq technology advancing toward a cure for cerebral cavernous malformations. Gain insights into scalable technologies and biological advances that are paving the way for treatments and cures for patients with rare genetic diseases, presented by leading researchers at the Broad Institute's annual symposium bringing together scientists, physicians, patients, patient-led foundations, and industry advisors.
