Kymriah – tisagenlecleucel<\/a><\/p>\n\n\n\nTisagenlecleucel, marketed as Kymriah, is a treatment for B-cell acute lymphoblastic leukemia (ALL) which uses the body’s own T cells to fight cancer (adoptive cell transfer).<\/p>\n\n\n\n
T cells from a person with cancer are removed, genetically engineered to make a specific chimeric cell surface receptor with components from both a T-cell receptor and an antibody specific to a protein on the cancer cell, and transferred back to the person.<\/p>\n\n\n\n
The T cells are engineered to target a protein called CD19<\/strong> that is common on B cells. A chimeric T cell receptor (“CAR-T”) <\/strong>is expressed on the surface of the T cell.<\/p>\n\n\n\nDisadvantages<\/strong><\/p>\n\n\n\n- Short-lived nature [Cells \u00e0 Long-lived and stable]<\/li>
- Immune response [Foreign body response]<\/li>
- Problem with viral vectors \u00e0 [Toxicity\/Immune response\/ Targeting issues]<\/li>
- Multi-genic disorders<\/li>
- Insertional mutagenesis [Wrong DNA integration] – Leukemia due to rapid T-cell proliferation<\/strong><\/li><\/ul>\n\n\n\n
Pricing Issues<\/strong><\/p>\n\n\n\n- Market price of Glybera<\/em>: US$ 1.1 million per treatment!!!<\/li>
- Cost of preclinical development, manufacturing and distribution<\/li>
- Personalised manufacturing and distribution<\/li><\/ul>\n\n\n\n
Ethical Considerations<\/strong><\/p>\n\n\n\n- Deciding whether somatic gene therapy is more or less ethical than germ-line gene therapy
- Death of Jesse Gelsinger, an 18-year-old boy (Ornithine transcarbamylase deficiency)<\/em><\/li><\/ul>
- Died from multiple organ failure caused by a severe immune reaction in response to the use of first-generation adenoviral vectors<\/li><\/ul><\/li><\/ul>\n\n\n\n
- Gene therapies use altered genes and potentially dangerous viruses, should treatments be tested more extensively?<\/li>
- Will the high costs of gene therapy make it available only to the wealthy?<\/li>
- Should people be allowed to use gene therapy to enhance basic human traits such as intelligence, or athletic ability?<\/strong><\/li><\/ul>\n\n\n\n
Gene Doping<\/strong><\/p>\n\n\n\nGene therapy for restoring muscle lost to age or disease is poised to enter the clinic, but athletes are eyeing it to enhance performance. The non-therapeutic use of cells, genes, genetic elements, or of the modulation of gene expression, having the capacity to improve athletic performance is defined as Gene Doping by the World Anti-Doping Agency (WADA).<\/p>\n\n\n\n
For example, gene doping could be used to provide athletes a source of erythropoietin (EPO), a hormone that promotes the formation of red blood cells that is already widely abused in sports.<\/p>\n\n\n\n
Another candidate gene is Insulin-like Growth Factor 1 (IGF-1) which partly controls the building and repair of muscles by stimulating the proliferation of satellite cells. <\/p>\n","protected":false},"excerpt":{"rendered":"
Gene therapy Gene therapy is an experimental treatment that involves introducing genetic material into a person\u2019s cells to fight or prevent disease. Approaches: Replacing a mutated gene that causes disease with a healthy copy of the gene. Inactivating, or \u201cknocking out,\u201d a mutated gene that is functioning improperly. Introducing a new gene into the body to help fight[…]\n","protected":false},"author":3,"featured_media":0,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_seopress_robots_primary_cat":"","_seopress_titles_title":"","_seopress_titles_desc":"","_seopress_robots_index":"","footnotes":""},"categories":[3],"tags":[],"_links":{"self":[{"href":"https:\/\/medicineplexus.com\/wp-json\/wp\/v2\/posts\/494"}],"collection":[{"href":"https:\/\/medicineplexus.com\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/medicineplexus.com\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/medicineplexus.com\/wp-json\/wp\/v2\/users\/3"}],"replies":[{"embeddable":true,"href":"https:\/\/medicineplexus.com\/wp-json\/wp\/v2\/comments?post=494"}],"version-history":[{"count":0,"href":"https:\/\/medicineplexus.com\/wp-json\/wp\/v2\/posts\/494\/revisions"}],"wp:attachment":[{"href":"https:\/\/medicineplexus.com\/wp-json\/wp\/v2\/media?parent=494"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/medicineplexus.com\/wp-json\/wp\/v2\/categories?post=494"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/medicineplexus.com\/wp-json\/wp\/v2\/tags?post=494"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}