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Tefidsogene civaparvovec, formerly known as SB-913, is an investigational therapy for genome editing. It consists of an adeno-associated virus (AAV) type 2/6 vector and a zinc finger nuclease (ZFN) system that inserts a correct copy of the Iduronate 2-Sulfatase (IDS) gene into the albumin locus in hepatocytes. Developed by Sangamo Therapeutics, tefidsogene civaparvovec is being investigated in mucopolysaccharidosis type II.
Tefidsogene civaparvovec, formerly known as SB-913, is an investigational therapy for genome editing. It consists of an adeno-associated virus (AAV) type 2/6 vector and a zinc finger nuclease (ZFN) system that inserts a correct copy of the Iduronate 2-Sulfatase (IDS) gene into the albumin locus in hepatocytes. Developed by Sangamo Therapeutics, tefidsogene civaparvovec is being investigated in mucopolysaccharidosis type II.
每项发明一条，涵盖美国、欧洲、PCT 与中国的同族专利。
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