Nucleic acid construct for treating hereditary coagulation factor deficiency and use thereof
AssigneeKanglin Biotech (Hangzhou) Co., Ltd.
InventorsHaoquan WU, Lingling SU, Yanfu DING, Xiaobin FU
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Abstract
A nucleic acid construct for treating hereditary coagulation factor deficiency and a novel use thereof are provided. The construct includes the following elements: a polynucleotide encoding coagulation factor IX; and an AAV cis-regulatory element. An adeno-associated virus prepared from the nucleic acid construct and a nucleic acid construct carrying a coagulation factor target gene may have a muscle targeting property in an intramuscular injection delivery mode, and help express the coagulation factor at a therapeutically effective amount.
Claims25
A nucleic acid construct, comprising the following elements:
- a polynucleotide coding a coagulation factor IX;
- and
- an adeno-associated virus (AAV) cis-regulatory element, comprising one or more selected from the group consisting of an inverted terminal repeat (ITR), a promoter, a kozak sequence, a simian vacuolating virus 40 (SV40) polyA, an untranslated region (UTR), and a post-transcriptional regulatory element (WPRE).
(canceled)
A method of treating a hereditary coagulation factor deficiency, comprising administering an AAV vector serotype targeting muscle tissues to a subject.
A nucleic acid construct, wherein an adeno-associated virus prepared from the nucleic acid construct has a muscle targeting property and expresses a coagulation factor at a therapeutically effective amount;
- and the nucleic acid construct has the muscle targeting property under an intramuscular injection delivery mode;
- wherein the nucleic acid construct comprises a vector backbone expressing an adeno-associated virus capsid protein, wherein the vector backbone is KL-pAAV-RC1, KL-pAAV-RC2, KL-pAAV-RC6, KL-pAAV-RC6.2FF, KL-pAAV-RC7, KL-pAAV-RCrh74, KL-pAAV-RC8, or KL-pAAV-RC9, having the nucleotide sequences separately shown in SEQ ID NOS:
- 25-27, 9, and 28-31 in sequence.
15-16. (canceled)
22-23. (canceled)
Dependencies are inferred from the claim text.