FIELD: biotechnology.
SUBSTANCE: present disclosure relates to nucleic acid molecules comprising a first inverted terminal repeat (ITR), a second ITR and a gene cassette coding a target sequence. In some embodiments, the target sequence encodes a miRNA and/or a therapeutic protein. In some embodiments, the therapeutic protein contains a blood coagulation factor, a growth factor, a hormone, a cytokine, an antibody, a fragment thereof and a combination thereof. In some embodiments, the first ITR and/or the second ITR are the ITR of a virus other than adeno-associated virus (AAV).
EFFECT: present disclosure relates to methods of treating a metabolic hepatic disorder in a subject, comprising administering to the subject a nucleic acid molecule or a polypeptide encoded therewith.
18 cl, 22 dwg, 16 tbl, 7 ex
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