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US 2020/0270635 A1Published application
Modulatory polynucleotides
AssigneeVoyager Therapeutics, Inc.
InventorsJinzhao Hou, Xin Wang, Pengcheng Zhou, Xiao-Qin Ren, Dinah Wen-Yee Sah
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Abstract
The present invention relates to adeno-associated viral (AAV) particles modulatory polynucleotides encoding at least one siRNA molecules and methods of use thereof.
Claims21
1-112. (canceled)
An adeno-associated virus (AAV) vector genome comprising a nucleic acid sequence positioned between two inverted terminal repeats (ITRs), wherein the nucleic acid sequence comprises a first polynucleotide and a second polynucleotide;
- wherein the first polynucleotide comprises a first region encoding a first sense strand sequence which comprises at least 15 nucleotides, and a second region encoding a first antisense strand sequence which comprises at least 15 nucleotides, and wherein the first sense strand sequence and the first antisense strand sequence share a region of complementarity of at least seven nucleotides in length;
- and wherein the second polynucleotide comprises a third region encoding a second sense strand sequence which comprises at least 15 nucleotides, and a fourth region encoding a second antisense strand sequence which comprises at least 15 nucleotides, and wherein the second sense strand sequence and the second antisense strand sequence share a region of complementarity of at least seven nucleotides in length.
Dependencies are inferred from the claim text.