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US 10,004,765 B2Granted
Dosage compensating transgenes and cells
AssigneeUniversity of Massachusetts
InventorsJeanne B. Lawrence, Jun Jiang, Lisa L. Hall
Life of this patent
Estimating the term…
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.
Claims15
A silencing vector comprising:
- a silencing element comprising a silencing sequence comprising SEQ ID NO:10 or SEQ ID NO:11 flanked by first and second targeting sequences, wherein the first and second targeting sequences target insertion of the silencing sequence into a sequence comprising SEQ ID NO:9 within intron 1 of the dual specificity tyrosine-phosphorylation-regulated kinase 1A (DYRK1A) gene;
- and
- a promoter operably linked to the silencing element.
Dependencies are inferred from the claim text.