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US 2016/0143951 A1Published application
Dosage compensating transgenes and cells
AssigneeUniversity of Massachusetts
InventorsJeanne B. Lawrence, Jun Jiang, Lisa L. Hall
Life of this patent
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Abstract
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.
Claims20
A silencing vector comprising:
- a silencing element comprising a silencing sequence flanked by first and second targeting sequences, wherein each of the first and second targeting sequences are homologous to at least 50 by in, or within 1 MB of, the dual specificity tyrosine-phosphorylation-regulated kinase 1A (DYRK1A) gene or the Regulator of calcineurin 1 (RCAN1) gene;
- and
- a promoter operably linked to the silencing element.
A silencing vector comprising:
- a silencing element comprising a silencing sequence flanked by first and second targeting sequences, wherein each of the first and second targeting sequences are homologous to at least 50 bp in, or within 1 MB of, the dual specificity tyrosine-phosphorylation-regulated kinase 1A (DYRK1A) gene or the Regulator of calcineurin 1 (RCAN1) gene;
- and
- a promoter operably linked to the silencing element.
A silencing vector comprising the sequence shown in SEQ ID NOs:
- 14, 16, 17, 18, 19, 20, 21, 22, or 23.
Dependencies are inferred from the claim text.