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Paper Details

Moving away from one disease at a time: Screening, trial design, and regulatory implications of novel platform technologies.
Am J Med Genet C Semin Med Genet
5
2023
patients, phosphomorpholinos, rare diseases, single-gene, small interfering RNAs
Author NameAffiliation
Amy BrowerAmerican College of Medical Genetics and Genomics
Aaron J GoldenbergCase Western Reserve University School of Medicine
Robert C GreenBroad Institute, Ariadne Labs and Harvard Medical School
Timothy W YuHarvard Medical School
Timothy W YuHarvard Medical School
Erika F AugustineKennedy Krieger Institute
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Release 1.2