摘要:
The invention provides an isolated and purified DNA set forth as SEQ ID NO:15 in the Sequence Listing and an antisense oligonucleotide complementary to the DNA. The DNA represents the splicing enhancer sequence (SES) in exon 45 of human dystrophin gene, and serves as a template in preparation of the antisense oligonucleotide, which is used to induce exon 45 skipping in certain group of patient with Duchenne muscular dystrophy to restore the reading frame of dystrophin mRNA.
摘要:
The invention provides an isolated and purified DNA set forth as SEQ ID NO:15 in the Sequence Listing and an antisense oligonucleotide complementary to the DNA. The DNA represents the splicing enhancer sequence (SES) in exon 45 of human dystrophin gene, and serves as a template in preparation of the antisense oligonucleotide, which is used to induce exon 45 skipping in certain group of patient with Duchenne muscular dystrophy to restore the reading frame of dystrophin mRNA.
摘要翻译:本发明提供了序列表中SEQ ID NO:15所示的分离和纯化的DNA,以及与DNA互补的反义寡核苷酸。 DNA代表人肌营养不良蛋白基因外显子45中的剪接增强子序列(SES),并且用作制备反义寡核苷酸的模板,其用于在某些具有杜氏肌营养不良症的患者群中诱导外显子45跳跃以恢复 阅读框肌营养不良蛋白mRNA。
摘要:
The present invention provides a chimera gene as constructed by fusing the gene for the extracelluar region of human growth hormone receptor with the genes for the transmembrane and cytoplasmic regions of thrombopoietin receptor, an expression plasmid for the said gene and animal cells capable of expressing the protein encoded by the said gene. The human-growth-hormone dependent animal cell line transformed with an expression plasmid for the said gene are useful in screening hormones which show affinity for the chimera receptor, as well as their agonists and antagonists.
摘要:
The present invention provides materials capable of screening human melatonin which comprise an animal cell containing an expression plasmid for the gene encoding a human melatonin receptor protein and having said protein expressed therein, as well as a method for screening the same, thus permitting not only human melatonin but also hormones showing affinity for the same, and their agonists or antagonists to be screened with an enhanced degree of precision.
摘要:
According to the present invention, there are provided apoptotic agents for tumor cells of the nervous system which comprise a vector having the γ 1 34.5 gene deleted from the genome of a herpes simplex virus. The vectors are able to cause infection in tumor cells of the nervous system, thereby inducing apoptosis, and also enable virus derivatives with enhanced thymidine-kinase sensitivity to be prepared, thus opening up a way for utilizing a drug for gene therapy.