hnRNP A1 基因剔除之動物模式及其用途hnRNP A1 KNOCKOUT ANIMAL MODEL AND USE THEREOF

專利類型

發明

專利國別 (專利申請國家)

美國

專利申請案號

14/092,678

專利證號

US 8,946,503 B2

專利獲證名稱

hnRNP A1 基因剔除之動物模式及其用途hnRNP A1 KNOCKOUT ANIMAL MODEL AND USE THEREOF

專利所屬機關 (申請機關)

高雄醫學大學

獲證日期

2015/02/03

技術說明

本案揭露hnRNP A1同基因型或異基因型的基因缺失的非人類哺乳動物，特別指老鼠，及此動物的組織及細胞。hnRNP A1是一個RNA結合蛋白質而且參與mRNA剪接的過程，mRNA剪接的缺失已經陸續發現與許多人類遺傳性疾病有關，如脊髓肌肉萎縮症(SMA)、裘馨氏肌肉萎縮症(DMD)、神經退化性疾病。hnRNP A1也已知在非小細胞肺癌、大腸直腸癌與急性白血病等癌細胞中大量表現。若能針對hnRNP A1參與這些疾病的機制深入研究或許可以開發出相關疾病的治療藥物。而藥物開發過程，動物模式是不可或缺的工具。本發明提供hnRNP A1基因剔除哺乳動物，包含hnRNP A1蛋白質表現量減少的異基因型哺乳動物。這些動物及其衍生之細胞將可以應用於相關疾病的治療藥物開發。 Disclosed are a non-human mammal, especially a mouse, with homozygous or heterozygous for defect of the hnRNP A1 gene, and a tissue and a cell of the non-human mammal. hnRNP A1 is a binding protein that associate with the process of mRNA splicing. Defects in mRNA splicing have emerged as a mechanism of many human genetic diseases, such as spinal muscular atrophy, Duchenne muscular dystrophy, neurodegenerative diseases. hnRNP A1 also has been reported upregulated in the cells of many cancers, such as non-small cell lung cancer, colorectal cance and acute leukemia. To understand the effects of hnRNP A1 may help to develop potential treatments for such patients. Moreover, an animal model is necessary during the process of drug development. This invention provides a hnRNP A1 knockout mammal, including a heterozygous mammal with low hnRNP A1 protein level. These animals and the cells derived from these animals can be used for the development of therapeutic drugs for related diseases.

備註

連絡單位 (專責單位/部門名稱)

智財保護與科技管理組

連絡電話

07-3138030

網址