Dosage compensating transgenes and cells

申请号 US14776672 申请日 2014-03-14 公开(公告)号 US10004765B2 公开(公告)日 2018-06-26
申请人 University of Massachusetts; 发明人 Jeanne B. Lawrence; Jun Jiang; Lisa L. Hall;
摘要 Methods and compositions for reducing expression of genes on Chromosome 21 (“Chr 21”) by targeting an XIST transgene to the Dual specificity tyrosine-phosphorylation-regulated kinase 1A (DYRK1A) gene or a Regulator of calcineurin 1 (RCAN1) gene, and cells and transgenic animals comprising an XIST transgene inserted into a DYRK1A or RCAN1 allele, e.g., cells and animals trisomic for human Chr 21 and mouse Chr 16.
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