OLIGONUCLEOTIDES FOR DIRECTED GENE KNOCKOUT

用于定向基因敲除的寡核苷酸

• 批准号: 6015332
• 负责人: MUTHIAH MANOHARAN
• 金额: $ 22.47万
• 依托单位: IONIS PHARMACEUTICALS, INC.
• 依托单位国家: 美国
• 财政年份: 1999
• 资助国家: 美国
• 起止时间: 1999-08-01 至 2002-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6015332
• 关键词: cell line; gene targeting; nucleic acid chemical synthesis; oligonucleotides; synthetic nucleotide; technology /technique development; transfection /expression vector; triple helix

DESCRIPTION (Adapted from the application): In this Phase I grant application, the researchers propose to synthesize cationic and neutral oligonucleotides to enhance the binding affinity of synthetic oligonucleotides capable of sequence specific triple helix formation which can serve as reagents for directed gene knockout. Triplex-forming oligonucleotides (TFOs) have been shown to inhibit transcription and induce site-specific mutagenesis and recombination in mammalian cells. These reagents can be used as tools for genetic manipulation and have the potential to be developed into gene based therapeutics for cancer, viral infections, and inflammatory diseases. The applicants suggest that the efficiency and binding affinity of triplex targeting can be significantly improved by novel modifications of the oligonucleotides that have been developed in the researcher's laboratories. PROPOSED COMMERCIAL APPLICATION: NOT AVAILABLE

描述（根据应用程序改编）：在此阶段I授予应用程序， 研究人员建议将阳离子和中性寡核苷酸合成为 增强能够序列的合成寡核苷酸的结合亲和力 特定的三重螺旋形成，可以用作定向基因的试剂 昏死。三核苷酸（TFO）已显示出抑制 转录和诱导位点特异性诱变和重组 哺乳动物细胞。这些试剂可以用作基因操纵的工具 并有可能发展成基于基因的癌症的疗法， 病毒感染和炎症性疾病。申请人建议 三重靶向的效率和结合亲和力可以显着 通过已有的新型寡核苷酸改进 在研究人员的实验室中开发。 拟议的商业应用程序：不可用