Bilaketaren emaitzak - Raymond Y. Wang

A Newborn Screening, Presymptomatically Identified Infant With Late-Onset Pompe Disease: Case Report, Parental Experience, and Recommendations nork Raymond Y. Wang

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Base editing rescues acid α-glucosidase function in infantile-onset Pompe disease patient-derived cells nork Chloe L. Christensen, Shih-Hsin Kan, Perla Andrade-Heckman, Allisandra K. Rha, Jerry F. Harb, Raymond Y. Wang

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Base editing corrects the common Salla disease SLC17A5 c.115C>T variant nork Jerry F. Harb, Chloe L. Christensen, Shih-Hsin Kan, Allisandra K. Rha, Perla Andrade-Heckman, Laura Pollard, Richard Steet, Jeffrey Y. Huang, Raymond Y. Wang

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