| abstract |
The disclosure provides a nucleic acid molecule comprising a first inverted terminal repeat (ITR), a second ITR, and a genetic cassette encoding a miRNA and / or a therapeutic protein. In certain embodiments, the therapeutic protein comprises a coagulation factor, such as a FVIII polypeptide, a FIX polypeptide, or a fragment thereof. In some embodiments, the first ITR and / or the second ITR is an ITR of a non-adeno-associated virus (AAV). This disclosure also provides a method of treating a bleeding disorder such as hemophilia, which comprises administering to the subject the nucleic acid molecule or a polypeptide encoded thereby. |