DEVELOPMENT OF ADENOVIRAL PROTEIN/DNA CONJUGATE VECTORS

腺病毒蛋白/DNA缀合物载体的开发

• 批准号: 2867988
• 负责人: NORIYUKI KASAHARA
• 金额: $ 4.48万
• 依托单位: GENETIC BIOTECHNOLOGY VENTURES, INC.
• 依托单位国家: 美国
• 财政年份: 1999
• 资助国家: 美国
• 起止时间: 1999-05-10 至 2000-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/2867988
• 关键词: Adenoviridae; gene targeting; gene therapy; heregulin; neoplastic cell; recombinant proteins; technology /technique development; transfection; transfection /expression vector; virus DNA; virus protein

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

描述：（改编自申请人的摘要）基因递送载体， 提出利用病毒蛋白的独特特征， 基因传递系统这个系统的关键特征 （1）基因治疗的靶向能力;（2）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;（4） 缺乏病毒基因，因此，毒力;（5）增强的能力， 进入细胞并传递治疗基因。预计该系统将保留 病毒载体的一些最好的特性，同时利用 病毒进化出的高效进入细胞的机制。该系统 预期通过引入细胞类型特异性 瞄准能力。由于该系统本质上是非病毒性的， 生物制剂固有的问题，从而使药品质量 控制最后的准备工作。该系统的潜在应用 包括用于癌症治疗的治疗基因或药物的靶向递送 或基因替代疗法。该模型系统使用调蛋白作为靶向 乳腺癌的配体，单纯疱疹胸苷激酶作为 治疗基因 拟议商业应用：不可用