DEVELOPMENT OF ADENOVIRAL PROTEIN/DNA CONJUGATE VECTORS

描述：(改编自申请者的摘要)基因运送工具是 提出了一种利用病毒蛋白的独特功能在靶向、非病毒 基因输送系统。这一系统的主要特点将有助于它们自己 基因治疗有(1)导弹般的靶向能力；(2)对基因治疗的能力 运输DNA，因此，治疗基因；(3)缺乏大小 限制，因此，携带任何大小基因的潜在能力； 缺乏病毒基因，因此缺乏毒力；(5)增强了 进入细胞并传递治疗性基因。预计该系统将保留 病毒载体的一些最佳特性，同时利用 病毒进化为有效进入细胞的机制。这个系统是 希望通过引入特定于细胞类型的方法来改进这些机制 目标定位能力。因为该系统本质上是非病毒的，所以它避免了 生物制剂固有的问题，从而使药品质量 对最后的准备工作进行控制。该系统的潜在应用前景 包括针对癌症治疗的治疗基因或药物的靶向输送 或者基因替代疗法。该模型系统使用heregin作为靶点 乳腺癌的配体，单纯疱疹病毒胸苷激酶作为 治疗性基因。 建议的商业应用：不可用

DESCRIPTION: (adapted from applicant's abstract) A gene delivery vehicle is proposed that uses unique features of viral proteins in a targeted, nonviral gene delivery system. The key features of this system that will lend themselves to gene therapy are (1) the missile-like targeting capacity; (2) the capacity to transport DNA, and, therefore, therapeutic genes; (3) the lack of size restriction and, therefore, the potential capacity to carry any size gene; (4) the lack of viral genes and, therefore, virulence; (5) the enhanced ability to enter cells and deliver therapeutic genes. The system is expected to retain some of the best properties of viral vectors while taking advantage of mechanisms that viruses have evolved to efficiently enter cells. This system is expected to improve these mechanisms by introducing cell type-specific targeting capabilities. Because the system is nonviral in nature, it avoids problems inherent in biologic agents, thus enabling pharmaceutical quality control over the final preparations. Potential application of this system includes targeted delivery of therapeutic genes or drugs for cancer treatment or gene replacement therapy. The model system uses heregulin as a targeting ligand for breast cancer, and herpes simplex thymidine kinase as the therapeutic gene. PROPOSED COMMERCIAL APPLICATION: NOT AVAILABLE