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US 2019/0169616 A1Published application
Modulatory polynucleotides
AssigneeVoyager Therapeutics, Inc.
InventorsDinah Wen-Yee Sah, Jinzhao Hou, Mathieu Nonnenmacher
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Abstract
The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of modulatory polynucleotides.
Claims28
A modulatory polynucleotide comprising
- (a) a stem and loop to form a stem-loop structure, the sequence of said stem-loop structure from 5′ to 3′ comprising:
- (i) a 5′ stem arm comprising a passenger strand;
- (ii) a loop region, wherein the loop region comprises a sequence selected from the group consisting of SEQ ID NO:
- 16, 10, 11, 12, 13, 14, 15, 17, 18, and 19;
- (iii) a 3′ stem arm comprising a guide strand, wherein a uridine is present at the 5′ end of the guide strand;
- (b) a first flanking region located 5′ to said stem-loop structure, wherein the first flanking region comprises a sequence selected from the group consisting of SEQ ID NO:
- 5, 1, 2, 3, 4, 6, 7, 8, and 9;
- and
- (c) a second flanking region located 3′ to said stem-loop structure and wherein said second flanking region comprises a sequence is selected from the group consisting of SEQ ID NO:
- 21, 20, 22, 23, 24, 25, 26 and 27.
7 more elements
A modulatory polynucleotide comprising
- (a) a stem and loop to form a stem-loop structure, the sequence of said stem-loop structure from 5′ to 3′ comprising:
- (i) a 5′ stem arm comprising a guide strand, wherein a uridine is present at the 5′ end of the guide strand;
- (ii) a loop region, wherein the loop region comprises a sequence selected from the group consisting of SEQ ID NO:
- 16, 10, 11, 12, 13, 14, 15, 17, 18, and 19;
- (iii) a 3′ stem arm comprising a passenger strand;
- (b) a first flanking region located 5′ to said stem-loop structure, wherein the first flanking region comprises a sequence selected from the group consisting of SEQ ID NO:
- 5, 1, 2, 3, 4, 6, 7, 8, and 9;
- and
- (c) a second flanking region located 3′ to said stem-loop structure and wherein said second flanking region comprises a sequence is selected from the group consisting of SEQ ID NO:
- 21, 20, 22, 23, 24, 25, 26 and 27.
7 more elements
The modulatory polunucleotide of 1 or 2, wherein the second flanking region comprises SEQ ID NO:
- 21.
Dependencies are inferred from the claim text.