genetherapy-deepwater：基因療法-深水-文庫(kù)吧
2024-12-28 08:03 本頁(yè)面
【正文】 lood stream to the bone marrow. Germline Gene Therapy ? Gene therapy in germline cells has the potential to affect not only the individual, but also their children as well. ? Any geic changes in the reproductive cells or changes made to the embryo before the stage of differentiation, would affect all future offspring of that person. This makes an vital distinction, affecting major ethcial issues Delivery of Genes ? Need an efficient method to deliver the gene to live cells. ? Done via vectors (gene carriers), delivering therapeutic genes to cells mon vector is viruses ? Alternative delivery methods seen with use of lipids and proteins Use of Viruses ? Viruses evolved a way of delivering their genes to human cells in pathogenic matter. ? Take advantage of this, manipulate genome to remove disease genes and insert therapeutic genes Risks with GT trials ? Viruses can usually infect more than one type of cell. ? When DNA is injected directly into a tumor, there is a slight chance that this DNA could unintentionally be introduced into reproductive cells. ? Other concerns include the possibility that transferred genes could be overexpressed Add a new chromosome? ? Experimentation seen with introduction of 47th chromosome ? Exist along 46th chromosome, large vector capable carrying geic code, that hopefully the immune system would not attack Understanding? ? Scientists know little about function of the thousands of genes. Attempting therapy without this knowledge would address few genes of a particular disease ? . Sickle Cell Anemia Sickle Cell Anemia ? Caused by error in gene that informs our body on how to make hemoglobin ? Prevalent in African Americans ? However deadly the error is, this increased the survival rate for those who also had malaria in the region Hurdles… ? Most geic disorders involve more than one gene, only few geic diseases involve merely one ? This multigene disorders also involve the environment, such as diet, exercise, smoking, etc. Everything has a price ? High costs associated with developing this technology, and regulations that needed to be implied on it with experimentations are great hurdles for
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