A Framework for Individualized Splice-Switching Oligonucleotide Therapy

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