遗传zy知识要点.pptxVIP

知 识 要 点遗传病治疗 干预方案遗传病治疗 – gene therapyGene therapyDefinitionStill in the progress of researchClasses: in vivo, ex vivoMethod of delivering DNAVirus and charactristicsAdenovirusAdeno-associated virusRetrovirusHerpes simplex virusLipisomeNaked DNAVector TransfectionTargeted gene therapy may result since some viruses infect certain body cells Adenoviruses（腺病毒）infect both dividing non-dividing cells effectivelyAdeno-Associated viruses do not cause illness in humans, can infect a wide variety of cells, integrate 95% of time in same locationRetroviruses （逆转录病毒）are of interest because they insert DNA into the genome of host where it remains permanently (integration), but often, randomlyVector TransfectionHerpes virus (HSV-1) strain primarily affects central nervous system (CNS)May help develop treatments for Alzheimer’s, Parkinson’s, and other genetic neurodegenerative diseasesHowever, although viral vectors may help, most human cells are not easily transfectedAlternative delivery Liposomes脂质体are small diameter, hollow particles made of lipid molecules Packaged with genes and injected into tissues A gene gun could also be used “Naked” DNA injected directly into body tissues (ex. effective in liver/muscle), but not enough cells express gene to have affect Artificial chromosomes may also deliver therapeutic geneNon-protein coding DNA with therapeutic gene Similar construction to normal chromosomes (designed for permanent incorporation)遗传病治疗 - RNAIncrease gene expression药物 – 降低甲基化 – 升高表达例子：地西他滨治疗镰形细胞贫血和地中海贫血Reducing the expressionRNA interference反向RNA – 降解遗传病治疗 – 蛋白质Dietary restriction - PKUReplacementTo cellular organelleEx cellularDiversion – urea cycle defectInhibition – hypercholesterolemia (part)Depletion - HemochromatosisAugmentation - Homocystinuria遗传病治疗 – 临床表型手术：先天性心脏病唇腭裂矫形先天性马蹄内翻足遗传病治疗 – 移植种类：器官移植肝细胞移植问题：排斥成瘤免疫抑制遗传病治疗发展现状高速发展，但总体并不令人满意。主要问题：一些药物只能在短期内缓解症状，但随着患者生存期的增加，新兴症状不断出现。一些药物在短期治疗中无副作用，一旦长期服用边可导致严重不良后果。治疗必须以准确及时的诊断为前提。肿瘤遗传学肿瘤遗传学癌基因定义原癌基因抑癌基因定义分类抑制生长，促进分化原癌基因（pr