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US 11,298,410 B2Granted
Methods and compositions for RNA-guided treatment of HIV infection
AssigneeTemple University of the Commonwealth System of Higher Education
InventorsKamel Khalili, Wenhui Hu, Yonggang Zhang
Life of this patent
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Abstract
Compositions for specifically cleaving target sequences in retroviruses include nucleic acids encoding a Clustered Regularly Interspace Short Palindromic Repeat (CRISPR) associated endonuclease and a guide RNA sequence complementary to one or more target nucleic acid sequences in a retrovirus genome.
Claims16
A composition comprising:
- (a) a first nucleic acid sequence encoding a Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)-associated Cas9 endonuclease;
- (b) a second nucleic acid sequence encoding a first guide RNA (gRNA), the first gRNA being complementary to a first target sequence within a 5′ long terminal repeat (LTR) or a 3′ LTR of a retroviral sequence;
- (c) a third nucleic acid sequence encoding a second gRNA, the second gRNA being complementary to a second target sequence within a GagD region of the retroviral sequence;
- and
- (d) an excipient.
2 more elements
A pharmaceutical composition comprising:
- (a) an expression vector comprising:
- (i) a first nucleic acid sequence encoding a Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)-associated Cas9 endonuclease;
- (ii) a second nucleic acid sequence encoding a first guide RNA (gRNA), the first gRNA being complementary to a first target sequence within a 5′ long terminal repeat (LTR) or a 3′ LTR of a retroviral sequence;
- and
- (iii) a third nucleic sequence encoding a second gRNA, the second gRNA being complementary to a second target sequence within a GagD region of the retroviral sequence;
- and
- (b) a pharmaceutically acceptable excipient.
4 more elements
An expression vector comprising:
- (a) a first nucleic acid sequence encoding a Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)-associated Cas9 endonuclease;
- (b) a second acid sequence encoding a first guide RNA (gRNA), the first gRNA being complementary to a first target sequence within a 5′ long terminal repeat (LTR) or a 3′ LTR of a retroviral sequence;
- (c) a nucleic acid sequence encoding a second gRNA, the second gRNA being complementary to a second target sequence within a GagD region of the retroviral sequence;
- and
- (d) a regulatory region.
2 more elements
A polynucleotide encoding:
- (a) a first nucleic acid sequence encoding a Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)-associated Cas9 endonuclease;
- (b) a second nucleic acid sequence encoding a first guide RNA (gRNA), the first gRNA being complementary to a first target sequence within a 5′ long terminal repeat (LTR) or a 3′ LTR of a retroviral sequence;
- and
- (c) a third nucleic acid sequence encoding a second gRNA, the second gRNA being complementary to a second target sequence within a GagD region of the retroviral sequence.
1 more element
Dependencies are inferred from the claim text.