FACTOR VIII SEQUENCES
    128.
    发明申请
    FACTOR VIII SEQUENCES 审中-公开
    因子VIII序列

    公开(公告)号:US20170049859A1

    公开(公告)日:2017-02-23

    申请号:US15254984

    申请日:2016-09-01

    Abstract: There is provided a nucleic acid molecule comprising a nucleotide sequence encoding for a functional factor VIII protein, wherein the portion of the nucleotide sequence encoding for the B domain of the factor VIII protein is between 90 and 111 nucleotides in length and encodes for an amino acid sequence comprising a sequence having at least 85% identity to SEQ ID NO: 4 and which comprises six asparagine residues. Also provided is a functional factor VIII protein, a vector comprising the above nucleic acid molecule, a host cell, a transgenic animal, a method of treating haemophilia, e.g. haemophilia A, and a method for the preparation of a parvoviral gene delivery vector.

    Abstract translation: 提供了包含编码功能性因子VIII蛋白的核苷酸序列的核酸分子,其中编码因子VIII蛋白的B结构域的核苷酸序列的部分长度在90和111个核苷酸之间,并编码氨基酸 序列包含与SEQ ID NO:4具有至少85%同一性的序列,其包含六个天冬酰胺残基。 还提供了功能性因子VIII蛋白,包含上述核酸分子的载体,宿主细胞,转基因动物,治疗血友病的方法,例如, 血友病A,以及用于制备细小病毒基因递送载体的方法。

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