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O19: Antisense Oligonucleotides Targeting Linked-Snps Provide Allele-Specific Knockdown of a Dominant-Negative SPTAN1 Pathogenic Variant in a Complex Genetic Region

Christiana Wang, Shenglan Li,Sen Zhao,Jefferson Sinson,Denise Lanza,Wei Wang,Gladys Zapata, Kristina Macakova, Nhi Ho, Blake Vuocolo,Gary Clark, Sandesh Nagamani, Lindsay Burrage, Jason Heaney, Huda Zoghbi, Matthew Rasband,Pengfei Liu

Genetics in Medicine Open（2025）

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