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P171: Developing an approach to screening rare genetic diagnoses for amenability to bespoke genetic therapy development by David Cheerie, Marlen Lauffer, Danique Beijer, Matthis Synofzik, Annemieke Aartsma-Rus, Michael Szego, Kimberly Amburgey, Brian Kalish, James Dowling, Gregory Costain
Published 2024-01-01
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