Tongyao LIU,Alexey SEREGIN,Robert T. PETERS,Jiayun LIU,Philip ZAKAS,Douglas DRAGER,Susannah PATARROYO-WHITE
申请号：
US16537192
公开号：
US20200069817A1
申请日:
2019.08.09
申请国别(地区):
US
年份:
2020
代理人:
摘要：
The present disclosure provides nucleic acid molecules comprising a first inverted terminal repeat (ITR), a second ITR, and a genetic cassette encoding a target sequence. In some embodiments, the target sequence encodes a miRNA and/or a therapeutic protein. In certain embodiments, the therapeutic protein comprises a clotting 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 is an ITR of a non-adeno-associated virus (AAV). The present disclosure also provides methods of treating a metabolic disorder of the liver in a subject comprising administering to the subject the nucleic acid molecule or a polypeptide encoded thereby.
