{"id":1484,"date":"2020-08-07T00:55:38","date_gmt":"2020-08-07T00:55:38","guid":{"rendered":"https:\/\/meddists.com\/learn\/pre-clinical\/medical-genetics\/genetic-diseases\/treatment-of-genetic-diseases-modern-therapies\/"},"modified":"2020-08-07T01:09:32","modified_gmt":"2020-08-07T01:09:32","slug":"treatment-of-genetic-diseases-modern-therapies","status":"publish","type":"page","link":"https:\/\/meddists.com\/learn\/pre-clinical\/medical-genetics\/genetic-diseases\/treatment-of-genetic-diseases-modern-therapies\/","title":{"rendered":"Treatment of Genetic Diseases: Modern Therapies"},"content":{"rendered":"\n<p class=\"wp-block-paragraph\"><div class=\"intro\">Modern therapies are those that help to influence and improve the phenotype by advanced methods.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">There are three main modern therapies:<\/p>\n\n\n\n<ol class=\"wp-block-list\"><li>Influencing the expression of the gene<\/li><li>Stem cell transplantation<\/li><li>Gene therapy<\/div><\/li><\/ol>\n\n\n<span class=\"block-heading\" id=\"header_1\">\n<h4 class=\"wp-block-heading\" class=\"wp-block-heading\" class=\"title_collection title1\">Influencing the expression of the gene<\/h4>\n<\/span><span class=\"block-content\" id=\"contents_1\">\n\n\n<ul class=\"wp-block-list\"><li>If the disease is caused by the over-production of a toxic protein eg Huntington disease, we can reduce the expression of that protein by targeting the RNA.<\/li><li>siRNA&#8217;s will do this by binding to the RNA<em>(details of this are discussed in the Medical Genetics I: Epigenetics)<\/em><\/li><\/ul>\n\n\n<\/span><span class=\"block-heading\" id=\"header_2\">\n<h4 class=\"wp-block-heading\" class=\"wp-block-heading\" class=\"title_collection title1\">Stem cell transplantation<\/h4>\n<\/span><span class=\"block-content\" id=\"contents_2\">\n\n\n<ul class=\"wp-block-list\"><li>This is when stem cells are used to produce the missing cells or tissue.<\/li><li>It has two forms: nuclear transplantation and hematopoeitic stem cell transplantation.<\/li><li>In <strong>nuclear transplantation<\/strong>, the donor nuclei(diploid nuclei) is put into an oocyte that its nucleus has been removed. This cloned embryo will go on to form a blastocyst that can be used to make several organs and tissues, or in some cases, can even be used to form a whole organism.<\/li><li>Because of this, it is understandable that it is not used because of several ethical and moral grounds; it involves the destruction of a human embryo by replacing the embryos DNA with the donor DNA.<\/li><li>In <strong>Hematopoeitic stem cell transplantation,&nbsp;<\/strong>the stem cells come from the placenta of already delivered babies because the placenta contains lots of embryonic pluripotent cells that can be used to form new cells.<\/li><li><strong>Bone marrow transplantation<\/strong> used for the treatment of hemoglobinopathies is also a form of Hematopoeitic stem cell transplantation. A suitable matching donor must be found to prevent graft rejection.<\/li><li>It has been successfully used in the treatment of leukaemias and lysosomal storage diseases. This is because the stem cells in the placenta are very immune tolerant and the probability of graft rejection is greatly reduced.<\/li><\/ul>\n\n\n<\/span><span class=\"block-heading\" id=\"header_3\">\n<h4 class=\"wp-block-heading\" class=\"wp-block-heading\" class=\"title_collection title1\">Gene therapy<\/h4>\n<\/span><span class=\"block-content\" id=\"contents_3\">\n\n\n<ul class=\"wp-block-list\"><li>This is a modern therapy that involves modification of the mutant gene by adding an extra sequence to it.<\/li><li>It can be ex vivo or in vivo gene therapy.<\/li><\/ul>\n\n\n\n<div class=\"wp-block-image\"><figure class=\"aligncenter\"><a href=\"https:\/\/meddists.com\/wp-content\/uploads\/2020\/03\/ExVivoGeneTherapy.jpg\" target=\"_blank\" title=\"Treatment of Genetic Diseases: Modern Therapies\"><img decoding=\"async\" src=\"https:\/\/meddists.com\/wp-content\/uploads\/2020\/03\/ExVivoGeneTherapy-600x451.jpg\" alt=\"\" class=\"wp-image-27445\"\/><\/a><figcaption><strong>Illustration and explanation of ex vivo gene therapy<\/strong>(Credit: Lizanne Koch, ChemBioDraw)<\/figcaption><\/figure><\/div>\n\n\n\n<div class=\"wp-block-image\"><figure class=\"aligncenter\"><a href=\"https:\/\/meddists.com\/wp-content\/uploads\/2020\/03\/In_vivo_gene_therapy.jpg\" target=\"_blank\" title=\"Treatment of Genetic Diseases: Modern Therapies\"><img decoding=\"async\" src=\"https:\/\/meddists.com\/wp-content\/uploads\/2020\/03\/In_vivo_gene_therapy.jpg\" alt=\"\" class=\"wp-image-27447\"\/><\/a><figcaption><strong>Illustration and explanation of in Vivo gene therapy<\/strong>(Credit: Lizanne Koch, ChemBioDraw)<\/figcaption><\/figure><\/div>\n\n\n\n<ul class=\"wp-block-list\"><li>It is banned for germ cells as it can be used to modify embryos but some countries permit it to be used for somatic cells.<\/li><li>It can be used to replace the missing gene or replace the mutant gene with the wild type one<\/li><li>The genes have to be introduced by viral methods(using vectors) or non-viral methods such as complementary DNA.<\/li><li>Vectors can be adenoviral vectors or retroviral vectors.<\/li><li>Adenoviral vectors can infect all cells, can be used for in vivo therapy and are introduced into the cells by <strong>Receptor-mediated endocytosis<\/strong>.<\/li><li>Retroviral vectors can only infect dividing cells eg cancer cells and cannot be used for in vivo gene therapy<\/li><\/ul>\n<\/span><div id=\"the_titles\" style=\"display:none;\"><h4 class=\"wp-block-heading\" class=\"wp-block-heading\">Influencing the expression of the gene<\/h4><h4 class=\"wp-block-heading\" class=\"wp-block-heading\">Stem cell transplantation<\/h4><h4 class=\"wp-block-heading\" class=\"wp-block-heading\">Gene therapy<\/h4><\/div>","protected":false},"excerpt":{"rendered":"<p>Influencing the expression of the gene If the disease is caused by the over-production of a toxic protein eg Huntington disease, we can reduce the expression of that protein by targeting the RNA. siRNA&#8217;s will do this by binding to the RNA(details of this are discussed in the Medical Genetics I: Epigenetics) Stem cell transplantation [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":0,"parent":1450,"menu_order":6,"comment_status":"closed","ping_status":"closed","template":"","meta":{"footnotes":""},"class_list":["post-1484","page","type-page","status-publish","hentry"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.8 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Treatment of Genetic Diseases: Modern Therapies &#8211; 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