脂小體帶正電荷的部分又會與細胞表面帶負電荷的唾液酸（sialic acid）.ppt

Overview onGene therapy Outline Introduction Gene silencing strategy Gene enhancing strategy Route for gene transfer Methods for gene transfer Application Projects hosted from our Lab 基因與疾病 人類的46個染色體內，有30億( 3x109 )個bp，共約有3萬個基因 約有5,000個人類的疾病是因為單個基因變化所引起。如：常見的sickle cell anemia、cystic fibrosis、Duchenne / Becker muscular dystrophy、thalassemia及phenylketonuria及不常見的severe combined immunodeficiency (SCID) 、Lesch-Nyhan syndrome及不同的脂肪(lipid) 及碳水化醣(carbohydrate)貯藏病。 最近10年來，也證實了所有腫瘤細胞的癌化也是由多種基因的突變、失落、轉移或擴增而引起。 治療角度 治療角度 「基因增強治療」 當原來的基因不健全，無法行使正常功能時，加入正常的基因來補其不足。 「基因置換治療」 移入正常的基因，經由細胞裡的 DNA重組，以取代原有的異常基因 「自殺基因治療」 當無法以單基因修復細胞功能時，考慮直接犧牲該細胞，特別針對癌症療法。 「基因沉默(silencing)治療」 Therapeutic point-of-view Gene silencing strategy Silencing strategy Anti-sense Ribozyme RNA decoy Dominant negative RNAi Ribozyme gene therapy RNAi DNA-direct RNAi Gene enhancing strategy Route for gene transfer Ex vivo in vivo gene therapy Ex vivo gene therapy introduces foreign genes in the laboratory into cells harvested from the body. The genetically modified cells are then administered back into the patient For in vivo gene therapy foreign genes are introduced directly into cells while the cells remain within patients. In vivo techniques treat cells in situ. History for Gene therapy It is only 40 years since the genetic code was deciphered, and only within the last 2 decades have scientists manipulated and transferred foreign genetic material into a cell, subsequently altering its phenotypic and functional characteristics. The initial human gene transfer trial in 1989 involved ex vivo gene therapy techniques to replace the defective hemoglobin gene with a normal gene in a patient with thalassemia. The initial in vivo gene therapy trial was done in 1992 Methods for gene transfer Retroviruses integrate the therapeutic gene into the genome of dividing target cells. the transferred gene becomes an inherited component of cells and may result in a permanent change in cell characteristics. limitation ge