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    • 94. 发明申请
    • METHODS OF GENE THERAPY FOR TREATING DISORDERS OF THE EAR BY ADMINISTERING A VECTOR ENCODING AN ATONAL-ASSOCIATED FACTOR
    • 通过管理编码相关因子的矢量来治疗耳朵疾病的基因治疗方法
    • US20140005257A1
    • 2014-01-02
    • US14024749
    • 2013-09-12
    • GENVEC, INC.
    • Douglas E. Brough
    • A61K38/17
    • A61K38/17A61K38/00A61K48/005A61P27/00C07K14/4702C12N7/00C12N15/86C12N2501/60C12N2710/10343
    • The invention is directed to a method of changing the sensory perception of an animal. The method comprises administering an expression vector comprising a nucleic acid sequence encoding an atonal-associated factor, which is expressed to produce the atonal-associated factor resulting in generation of hair cells that allow perception of stimuli in the inner ear. Also provided is a method of generating a hair cell in differentiated sensory epithelia in vivo. The method comprises contacting differentiated sensory epithelial cells with an adenoviral vector (a) deficient in one or more replication-essential gene functions of the E1 region and E4 region, (b) comprising a spacer in the E4 region, and (c) comprising a nucleic acid sequence encoding an atonal-associated factor. The nucleic acid sequence is expressed to produce the atonal-associated factor such that a hair cell is generated. An adenoviral vector encoding an atonal-associated factor also is provided.
    • 本发明涉及一种改变动物的感觉知觉的方法。 所述方法包括施用包含编码无关联因子的核酸序列的表达载体,所述核酸序列被表达以产生导致产生能够感知内耳中的刺激的毛细胞的无关性因子。 还提供了一种在体内在分化感觉上皮细胞中产生毛细胞的方法。 该方法包括使分化的感觉上皮细胞与E1区和E4区的一个或多个复制必需基因功能缺陷的腺病毒载体(a)接触,(b)在E4区中包含间隔基,和(c)包含 编码无关联因子的核酸序列。 表达核酸序列以产生无创因子,从而产生毛细胞。 还提供了编码无关联因子的腺病毒载体。