FIELD: biotechnology.
SUBSTANCE: invention can be used in medical practice. The invention discloses novel lentiviral vectors containing codon-optimized factor VIII sequences, as well as methods of using such lentiviral vectors. The invention can be used for gene therapy, wherein lentiviral gene delivery ensures stable integration of the transgene expression cassette into the genome of target cells (eg. liver cells, hepatocytes). The invention also relates to methods of treating bleeding disorders such as hemophilia (e.g., hemophilia A) comprising administering to a subject in need thereof a liver cell-targeted lentiviral vector containing a codon-optimized factor VIII nucleic acid sequence at low dosages (from 1*108 TE/kg or less to 1.5*1010 TE/kg).
EFFECT: use of lentiviral vectors expressing factor VIII.
28 cl, 13 ex, 19 tbl, 22 dwg
| Title | Year | Author | Number |
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