复旦大学：《医学遗传学 Medical Genetics》课程教学资源（PPT课件讲稿）19 遗传疾病的治疗（基因治疗）Gene Therapy

Medical Genetics 19基因治疗 Gene Therapy

Medical Genetics 19 基因治疗 Gene Therapy

Medical Genetics 1. What is gene therapy? Genes, which are carried on chromosomes, are the basic physical and functional units of heredity Genes are specific sequences of bases that encode instructions on how to make proteins

Medical Genetics 1. What is gene therapy? Genes, which are carried on chromosomes, are the basic physical and functional units of heredity. Genes are specific sequences of bases that encode instructions on how to make proteins

Medical Genetics Although genes get a lot of attention, it's the proteins that perform most life functions and even make up the majority of cellular structures

Medical Genetics Although genes get a lot of attention, it’s the proteins that perform most life functions and even make up the majority of cellular structures

Medical Genetics When genes are altered so that the encoded proteins are unable to carry out their normal functions genetic disorders can result

Medical Genetics When genes are altered so that the encoded proteins are unable to carry out their normal functions, genetic disorders can result

Medical Genetics Gene therapy is a technique for correcting defective genes responsible for disease development

Medical Genetics Gene therapy is a technique for correcting defective genes responsible for disease development

Medical Genetics Researchers may use one of several approaches for correcting faulty genes; A normal gene may be inserted into a nonspecific location within the genome to replace a nonfunctional gene. This approach is most common 2. An abnormal gene could be swapped for a norma gene through homologous recombination 3. The abnormal gene could be repaired through selective reverse mutation, which returns the gene to its normal function 4. The regulation(the degree to which a gene is turned on or off) of a particular gene could be altered

Medical Genetics Researchers may use one of several approaches for correcting faulty genes: 1. A normal gene may be inserted into a nonspecific location within the genome to replace a nonfunctional gene. This approach is most common. 2. An abnormal gene could be swapped for a normal gene through homologous recombination. 3. The abnormal gene could be repaired through selective reverse mutation, which returns the gene to its normal function. 4. The regulation (the degree to which a gene is turned on or off) of a particular gene could be altered

Medical Genetics 2. How does gene therapy work? In most gene therapy studies, a normal"gene is inserted into the genome to replace an abnormal disease-causing gene

Medical Genetics 2. How does gene therapy work? In most gene therapy studies, a "normal" gene is inserted into the genome to replace an "abnormal," disease-causing gene

Medical Genetics a carrier molecule called a vector must be used to deliver the therapeutic gene to the patient's target cells. Currently, the most common vector is a virus that has been genetically altered to carry normal human dna

Medical Genetics A carrier molecule called a vector must be used to deliver the therapeutic gene to the patient's target cells. Currently, the most common vector is a virus that has been genetically altered to carry normal human DNA

Medical Genetics Viruses have evolved a way of encapsulating and delivering their genes to human cells in a pathogenic manner

Medical Genetics Viruses have evolved a way of encapsulating and delivering their genes to human cells in a pathogenic manner

Medical Genetics Scientists have tried to take advantage of this capability and manipulate the virus genome to remove disease-causing genes and insert therapeutic genes

Medical Genetics Scientists have tried to take advantage of this capability and manipulate the virus genome to remove disease-causing genes and insert therapeutic genes