PubMed · The American Journal of Human Genetics· Olivia Kim-McManus·· 3 天前
诊断与检测：Scaling haplospecific antisense oligonucleotides from N-of-1 to broad use in genetic disease populations by diplotyping.
Scaling haplospecific antisense oligonucleotides from N-of-1 to broad use in genetic disease populations by diplotyping.
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原文摘录：Antisense oligonucleotides (ASOs) are versatile disease-modifying therapies for genetic diseases. An accelerated US Food and Drug Administration (FDA) pathway enables ASO treatment trial initiation in an individual within a year.
来源：PubMed · The American Journal of Human Genetics · pubmed.ncbi.nlm.nih.gov