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US 12,522,843 B2Granted
Compositions and methods of chromosomal silencing
AssigneeCarryGenes Bioengineering, LLC
InventorsEdward Perkins, Amy Greene, Dominique Broccoli
Life of this patent
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Abstract
The present invention encompasses compositions and methods to create a whole chromosome regulatable “kill” switch to inactivate expression of one or more therapeutic genes expressed from a synthetic chromosome.
Claims11
An autonomously-replicating, non-integrating, stable mammalian synthetic chromosome comprising:
- multiple copies of a site-specific unidirectional recombination site;
- an active mammalian centromere comprising amplified ribosomal DNA (rDNA);
- a regulatable expression cassette comprising a mammalian X inactivation center (Xic) encoding at least an X-inactive specific transcript (Xist) and product(s) from at least one gene of interest;
- and
- a first regulatable promoter controlling expression of Xist;
- wherein expression of Xist acts as a whole chromosome inactivation switch that controls silencing of the synthetic chromosome.
3 more elements
An autonomously-replicating, non-integrating, stable mammalian synthetic chromosome comprising:
- multiple copies of a site-specific unidirectional recombination site;
- at least one gene of interest;
- an active mammalian centromere comprising amplified ribosomal DNA (rDNA);
- a regulatable promoter;
- and
- operably linked to said promoter, a mammalian X inactivation center (Xic) encoding gene products necessary and sufficient for initiation of inactivation, thereby allowing reversibly regulatable silencing of expression from the synthetic chromosome.
3 more elements
Dependencies are inferred from the claim text.