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In vivo base editing gene therapy for heterozygous familial hypercholesterolemia: a phase 1 trial
Abstract Heterozygous familial hypercholesterolemia is a common genetic disorder characterized by lifelong elevation of serum low-density lipoprotein cholesterol (LDL-C) and premature atherosclerotic …
Integrated epidemiological and molecular data inform the relationship between precancer and cancer states of esophageal adenocarcinoma
Abstract Cancer generally takes years to evolve, and early diagnosis can prevent life-threatening cancer. Establishing a link between precancerous states and cancer is essential for effective screenin …
Introducing iCatalog as a clinical decision support tool for collaborative pediatric precision oncology studies
Abstract Background Next-generation sequencing (NGS) tests are integral to oncology care. To address the need for clinical and NGS data management, interpretation, and reporting, we developed iCatalog …
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