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US 2026/0109979 A1Published application
Nucleic acid regulatory elements for gene expression in the liver and methods of use
AssigneeMeiragtx UK II Limited
InventorsAlexandria Forbes, Josefa Sullivan, Chenjin Jin, Enrico Mossotto, Matthew During, Ce Feng Liu, Dustin Lee, Jessica Schmerler
Life of this patent
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Abstract
The application relates to nucleic acid regulatory elements (NAREs) that are able to enhance expression of genes in the liver. The application further relates to methods employing NAREs and uses of the NAREs. Expression cassettes and vectors containing NAREs are also disclosed. These are particularly useful for applications using gene therapy.
Claims87
A NARE comprising:
- (i) a sequence that is at least 90% identical to SEQ ID NO:
- 74;
- and (ii) a sequence that is at least 90% identical to any one of SEQ ID NOs:
- 193, 194, 207, 209-214, 216-232, 233-235, or 249.
1 more element
A NARE comprising:
- (i) a sequence that is at least 90% identical to SEQ ID NO:
- 150;
- and (ii) a sequence that is at least 90% identical to SEQ ID NO:
- 151 or SEQ ID NO:
- 155.
2 more elements
A NARE comprising:
- (i) a sequence that is at least 90% identical to any one of SEQ ID NOs:
- 154, 157, or 158;
- (ii) a sequence that is at least 90% identical to any one of SEQ ID NOs:
- 153, 156, 161, 162, 163, 166, 167, 168, or 174;
- and (iii) optionally, a sequence that is at least 90% identical to any one of SEQ ID NOs:
- 169, 172 or 173.
3 more elements
A NARE comprising:
- (i) a sequence that is at least 90% identical to SEQ ID NO:
- 153;
- (ii) a sequence that is at least 90% identical to SEQ ID NO:
- 171;
- and (iii) a sequence that is at least 90% identical to any one of SEQ ID NOs:
- 154, 157, 158 or 176.
3 more elements
A NARE comprising:
- (i) a sequence that is at least 90% identical to SEQ ID NO:
- 153;
- (ii) a sequence that is at least 90% identical to any one of SEQ ID NOs:
- 154, 157, 158, 176, 188, or 250;
- and (iii) optionally, SEQ ID NO:
- 155 or SEQ ID NO:
- 171.
4 more elements
A NARE comprising:
- (i) a sequence that is at least 90% identical to SEQ ID NO:
- 155;
- (ii) a sequence that is at least 90% identical to SEQ ID NO:
- 176 and (iii) a sequence that is at least 90% identical to any one of SEQ ID NOs:
- 175 or 177-187.
2 more elements
A NARE comprising:
- (i) a sequence that is at least 90% identical to SEQ ID NO:
- 190 or SEQ ID NO:
- 191;
- (ii) a sequence that is at least 90% identical to any one of SEQ ID NOs:
- 193, 194, or 195;
- and (iii) optionally, a sequence that is at least 90% identical to SEQ ID NO:
- 162;
- (iv) optionally, a sequence that is at least 90% identical to SEQ ID NO:
- 168;
- and (v) optionally, SEQ ID NO:
- 169.
8 more elements
A NARE comprising:
- (i) a sequence that is at least 90% identical to SEQ ID NO:
- 168;
- (ii) a sequence that is at least 90% identical to any one of SEQ ID NOs:
- 197, 202 or 203;
- (iv) optionally, a sequence that is at least 90% identical to any one of SEQ ID NOs:
- 190, 191, 192, 196, 202, 203, 204, 205 or 206;
- (v) optionally, a sequence that is at least 90% identical to SEQ ID NO:
- 155 or SEQ ID NO:
- 162;
- and (vi) optionally, a sequence that is at least 90% identical to any one of SEQ ID NOs:
- 166, 193, or 194.
8 more elements
A NARE comprising:
- (i) a sequence that is at least 90% identical to SEQ ID NO:
- 192;
- (ii) a sequence that is at least 90% identical to SEQ ID NO:
- 193 or SEQ ID NO:
- 194;
- and (iii) a sequence that is at least 90% identical to any one of SEQ ID NOs:
- 201-203.
4 more elements
A NARE comprising:
- (i) a sequence that is at least 90% identical to SEQ ID NO:
- 155;
- (ii) a sequence that is at least 90% to any one of SEQ ID NOs:
- 204, 205, 206, or 236-238;
- (iii) optionally, a sequence that is at 90% identical to SEQ ID NO:
- 168;
- (iv) optionally, a sequence that is at least 90% identical to SEQ ID NO:
- 169;
- and (v) optionally, a sequence that is at least 90% identical to SEQ ID NO:
- 197.
7 more elements
A NARE comprising:
- (i) a sequence that is at least 90% identical to SEQ ID NO:
- 248;
- and (ii) a sequence that is at least 90% identical to any one of SEQ ID NOs:
- 239-247.
1 more element
A NARE comprising:
- (i) a sequence that is at least 90% identical to SEQ ID NO:
- 208;
- and (ii) a sequence that is at least 90% identical to any one of SEQ ID NOs:
- 209-214.
1 more element
A NARE comprising:
- (i) a sequence that is at least 90% identical to SEQ ID NO:
- 193 or SEQ ID NO:
- 194;
- (ii) a sequence that is at least 90% identical to SEQ ID NO:
- 215;
- and (iii) a sequence that is at least 90% identical to any one of SEQ ID NOs:
- 207 or 209-215.
4 more elements
A NARE comprising:
- (i) a sequence that is at least 90% identical to SEQ ID NO:
- 155;
- (ii) a sequence that is at least 90% identical to SEQ ID NO:
- 205 or SEQ ID NO:
- 206;
- and (iii) a sequence that is at least 90% identical to any one of SEQ ID NOs:
- 207 or 209-215.
4 more elements
A NARE comprising:
- a) (i) a sequence that is at least 90% identical to SEQ ID NO:
- 53;
- b) (i) a sequence that is at least 90% identical to SEQ ID NO:
- 195;
- and (ii) a sequence that is at least 90% identical to SEQ ID NO:
- 202;
- c) (i) a sequence that is at least 90% identical to a sequence that is at least 90% identical to SEQ ID NO:
- 168;
- (ii) a sequence that is at least 90% identical to a sequence that is at least 90% identical to SEQ ID NO:
- 169;
- (iii) a sequence that is at least 90% identical to a sequence that is at least 90% identical to SEQ ID NO:
- 197;
- and (iv) a sequence that is at least 90% identical to a sequence that is at least 90% identical to SEQ ID NO:
- 203;
- d) (i) a sequence that is at least 90% identical to SEQ ID NO:
- 74;
- and (ii) a sequence that is at least 90% identical to a sequence that is at least 90% identical to SEQ ID NO:
- 220;
- e) (i) a sequence that is at least 90% identical to SEQ ID NO:
- 74;
- and (ii) a sequence that is at least 90% identical to a sequence that is at least 90% identical to SEQ ID NO:
- 229;
- or
- f) (i) a sequence that is at least 90% identical to SEQ ID NO:
- 74;
- and (ii) a sequence that is at least 90% identical to a sequence that is at least 90% identical to SEQ ID NO:
- 230.
24 more elements
A NARE comprising:
- (i) a sequence that is at least 90% identical to SEQ ID NO:
- 166 and (ii) a sequence that is at least 90% identical to any one of SEQ ID NOs:
- 154, 157, or 158.
A NARE comprising:
- (i) a sequence that is at least 90% identical to SEQ ID NO:
- 165 and (ii) a sequence that at least 90% identical to any one of SEQ ID NOs:
- 154, 157, or 158.
A NARE comprising:
- (i) a sequence that is at least 90% identical to SEQ ID NO:
- 199;
- and (ii) a sequence that is at least 90% identical to SEQ ID NO:
- 193 or SEQ ID NO:
- 194.
2 more elements
A NARE comprising:
- (i) a sequence that is at least 90% identical to SEQ ID NO:
- 164;
- and (ii) a sequence that is least 90% identical to SEQ ID NO:
- 200.
1 more element
A NARE comprising:
- (i) a sequence that is at least 90% identical to SEQ ID NO:
- 189 and (ii) a sequence that is at least 90% identical to SEQ ID NO:
- 193 or SEQ ID NO:
- 194.
1 more element
A NARE comprising:
- (i) a sequence that is at least 90% identical to SEQ ID NO:
- 202 or SEQ ID NO:
- 203;
- and (ii) a sequence that is at least 90% identical to SEQ ID NO:
- 195.
2 more elements
A nucleic acid regulatory element (NARE) comprising a sequence that is at least 90% identical to any one of SEQ ID NOs:
- 3-8, 11-69, 71-73, or 76-149.
An expression construct comprising the NARE of any one of the preceding claims and an operatively linked transgene.
A vector comprising a nucleic acid sequence comprising (i) the expression construct of 70, and (ii) one or more inverted terminal repeats (ITR).
Dependencies are inferred from the claim text.