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MOLECULAR BASIS OF CEREBRAL DEGENERATION

MOLECULAR BASIS OF CEREBRAL DEGENERATION
大脑退化的分子基础
批准号：
3393797
负责人：
JOHN S O'BRIEN
金额：
$16.92万
依托单位：
UNIVERSITY OF CALIFORNIA, SAN DIEGO
依托单位国家：
美国
项目类别：
财政年份：
1981
资助国家：
美国
项目状态：
已结题
起止时间：
1981-09-01 至 1991-11-30
来源：
https://reporter.nih.gov/project-details/3393797
关键词：
Sandhoff disease Tay Sachs disease affinity chromatography beta N acetylhexosaminidase beta galactosidase complementary DNA dogs embryo /fetus gangliosidosis GM1 gel electrophoresis gene expression gene therapy genetic library genetic manipulation genetic mapping genetic models human subject human tissue immunogenetics molecular cloning molecular pathology neurochemistry nucleic acid sequence prenatal diagnosis radioimmunoassay tissue /cell culture

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中文摘要
翻译
三个人类溶酶体基因，即β-半乳糖苷酶、己糖氨酸酶A和 己糖氨酸酶B，将被克隆。每个的cDNAs将从一个 抗体反应性筛选人肝lambda gtll文库 斑块。将分离并测序全长cDNAs以建立 与每种蛋白质的氨基酸序列共线性。与 GM1和GM2神经节苷脂沉积症的突变将在mRNA分析后进行 和基因组片段来确定选定的核酸缺陷 细胞系。β-半乳糖苷酶基因将被插入到一个表达中 将尝试对携带GM1的比格犬进行载体和基因治疗 神经节苷脂沉着症。
英文摘要
Three human lysosomal genes, beta-galactosidase, hexosaminidase A and hexosaminidase B, will be cloned. cDNA's for each will be isolated from a human liver lambda gtll library by screening for antibody reactive plaques. Full-length cDNA's will be isolated and sequenced to establish colinearity with amino acid sequences for each protein. Comparisons with mutants with GM1 and GM2 gangliosidosis will be made after analysis of mRNA and genomic fragments to establish the nucleic acid defects in selected cell lines. Beta-galactosidase cDNA will be inserted into an expression vector and gene therapy will be attempted in beagles with GM1 gangliosidosis.
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LYSOSOMAL ENZYMES
3 LYSOSOMAL ENZYMES
3 LYSOSOMAL ENZYMES
3 LYSOSOMAL ENZYMES
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