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US 2026/0248960 A1Published application
Systems for enhancing mrna expression and uses thereof
AssigneeThe Johns Hopkins University
InventorsJeffery Coller, Bahareh Torkzaban
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Abstract
Provided herein are systems comprising: (a) an RNA binding moiety; and (b) a gRNA hybrid comprising (i) a complementary region that hybridizes with an mRNA of an active allele of a gene associated with a disorder associated with a decrease in the expression of a protein from the mRNA and (ii) a poly(A) tail, wherein the gRNA hybrid forms a complex with the RNA binding moiety.
Claims34
A system comprising:
- (a) an RNA binding moiety;
- and
- (b) a gRNA hybrid comprising (i) a complementary region that hybridizes with an mRNA of an active allele of a gene associated with a disorder associated with a decrease in the expression of a protein from the mRNA and (ii) a poly(A) tail;
- wherein the gRNA hybrid forms a complex with the RNA binding moiety.
1 more element
A recombinant expression system comprising:
- (a) a nucleic acid sequence encoding an RNA binding moiety;
- and
- (b) a nucleic acid sequence encoding a gRNA hybrid comprising (i) a complementary region that hybridizes with an mRNA of an active allele of a gene associated with a disorder associated with a decrease in the expression of a protein from the mRNA and (ii) a poly(A) tail;
- wherein the gRNA hybrid forms a complex with the RNA binding moiety.
1 more element
Dependencies are inferred from the claim text.