A Framework for Individualized Splice-Switching Oligonucleotide Therapy
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https://zenodo.org/record/7783847
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This RNA-seq dataset was generated and analyzed as described in Kim, Woo, de Gusmao, Zhao et al, "A Framework for Individualized Splice-Switching Oligonucleotide Therapy" Nature 2023.
The RNA-seq dataset was obtained from cells derived from ataxia-telangiectasia (A-T) patients or their parents, and it comprises four groups:
(1) RNA-seq of fibroblasts from an A-T patient with c.7865C>T (proband) and her mother and father.
(2) RNA-seq of fibroblasts from an A-T patient with c.7865C>T (proband), transfected with ASOs (mock, NT-22, AT008, AT026).
(3) RNA-seq of whole blood from an A-T patient with c.5763-1050A>G (DDP_ATCP_42) and three other A-T patients without the variant (DDP_ATCP_96, DDP_ATCP_103, DDP_ATCP_151).
(4) RNA-seq of fibroblasts from an A-T patient with c.5763-1050A>G (DDP_ATCP_42), transfected with ASOs (naive, NT-22, AT043, AT056, AT057).
创建时间:
2023-07-10



