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

Bibliographic Details
Main Authors: Christiana Wang, Shenglan Li, Sen Zhao, Jefferson Sinson, Nhi Ho, Blake Vuocolo, Gary Clark, Sandesh Nagamani, Pengfei Liu
Format: Article
Language:English
Published: Elsevier 2024-01-01
Series:Genetics in Medicine Open
Online Access:http://www.sciencedirect.com/science/article/pii/S2949774424001638
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author Christiana Wang
Shenglan Li
Sen Zhao
Jefferson Sinson
Nhi Ho
Blake Vuocolo
Gary Clark
Sandesh Nagamani
Pengfei Liu
author_facet Christiana Wang
Shenglan Li
Sen Zhao
Jefferson Sinson
Nhi Ho
Blake Vuocolo
Gary Clark
Sandesh Nagamani
Pengfei Liu
author_sort Christiana Wang
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spelling doaj.art-3cfb569dd53c478ab149d90a8ea8cc5e2024-03-09T09:30:43ZengElsevierGenetics in Medicine Open2949-77442024-01-012101017O19: Antisense oligonucleotide targeting a linked-SNP provides allele-specific and effective knockdown to a dominant negative SPTAN1 pathogenic variantChristiana Wang0Shenglan Li1Sen Zhao2Jefferson Sinson3Nhi Ho4Blake Vuocolo5Gary Clark6Sandesh Nagamani7Pengfei Liu8Baylor College of MedicineBaylor College of MedicineBaylor College of MedicineBaylor College of MedicineBaylor College of MedicineBaylor College of MedicineBaylor College of Medicine, Texas Children's HospitalBaylor College of Medicine, Texas Children's HospitalBaylor College of Medicinehttp://www.sciencedirect.com/science/article/pii/S2949774424001638
spellingShingle Christiana Wang
Shenglan Li
Sen Zhao
Jefferson Sinson
Nhi Ho
Blake Vuocolo
Gary Clark
Sandesh Nagamani
Pengfei Liu
O19: Antisense oligonucleotide targeting a linked-SNP provides allele-specific and effective knockdown to a dominant negative SPTAN1 pathogenic variant
Genetics in Medicine Open
title O19: Antisense oligonucleotide targeting a linked-SNP provides allele-specific and effective knockdown to a dominant negative SPTAN1 pathogenic variant
title_full O19: Antisense oligonucleotide targeting a linked-SNP provides allele-specific and effective knockdown to a dominant negative SPTAN1 pathogenic variant
title_fullStr O19: Antisense oligonucleotide targeting a linked-SNP provides allele-specific and effective knockdown to a dominant negative SPTAN1 pathogenic variant
title_full_unstemmed O19: Antisense oligonucleotide targeting a linked-SNP provides allele-specific and effective knockdown to a dominant negative SPTAN1 pathogenic variant
title_short O19: Antisense oligonucleotide targeting a linked-SNP provides allele-specific and effective knockdown to a dominant negative SPTAN1 pathogenic variant
title_sort o19 antisense oligonucleotide targeting a linked snp provides allele specific and effective knockdown to a dominant negative sptan1 pathogenic variant
url http://www.sciencedirect.com/science/article/pii/S2949774424001638
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