O19: Antisense oligonucleotide targeting a linked-SNP provides allele-specific and effective knockdown to a dominant negative SPTAN1 pathogenic variant

Christiana Wang, Shenglan Li,Sen Zhao,Jefferson Sinson, Nhi Ho, Blake Vuocolo,Gary Clark,Sandesh Nagamani,Pengfei Liu

Genetics in Medicine Open(2024)

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