FIELD: biotechnology.
SUBSTANCE: group of inventions including a method of treating a person suffering from hemophilia A (variants) is described. In one embodiment, the method includes administering to a person having hemophilia A recombinant adeno-associated virus (rAAV) vector, wherein the vector genome includes a nucleic acid variant encoding factor VIII (FVIII) having a B-domain deletion (hFVIII-BDD), wherein the nucleic acid variant has 95% or greater identity with SEQ ID NO:7.
EFFECT: invention expands the arsenal of methods of treating hemophilia A leading to the absence of spontaneous bleeding in a person for at least 1 year.
66 cl, 28 dwg, 6 tbl, 10 ex
| Title | Year | Author | Number |
|---|---|---|---|
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|
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| RECOVERED NUCLEIC ACID WHICH CODES FVIII-BDD-BASED FUSION PROTEIN AND HETEROLOGOUS SIGNAL PEPTIDE, AND USE THEREOF | 2022 |
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|
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