Disadvantages Of Gene Therapy

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The Science Gene therapy : Gene therapy is an experimental technique that can be used in order to treat or prevent disease by targeting the patients genes directly through the use of a vector. Approaches to /uses of gene therapy: Replacing mutated or missing Gene- The most common approach to gene therapy is to replace a defective, mutated or missing gene with a functioning corrected copy in the patient's cells. For example replacing the damaged CFTR gene on chromosome 7 in order to treat cystic fibrosis. Gene silencing- This method is used to inactivate a gene that is not working properly so that no protein is made from it. Introducing new genes- This is when a new gene is introduced into the patient's cells for a specific purpose. For example…show more content…
The patient who receives the therapy is the only one affected. Germ line gene therapy: Targets the genes of the patients germ cells ( Sperm cells or egg cells). The therapy is heritable can be passed on to the patients offspring Delivering the gene: Inserting a gene directly in a patient's cells does not usually work and is very difficult and so vectors (carriers) are used instead. There are two main types of vectors viral and non-viral. Viral- Viruses are very useful as vectors do you to their ability to infect cells. In order to be used as vectors the genetic information that normal causes the disease is removed and replaced by the desired genes. There are different types that can be used for different purposes:…show more content…
When added to the cell they fuse with the cell membrane and the DNA is transferred to the cell. Does not integrate into the cell’s genome It has no maximum length of DNA. Does not target a specific cell. No immune response but are sometimes toxic + more suited for ex-vivo therapy (outside the body). Naked DNA-Does not integrate into the cell’s genome It has no maximum length of DNA. Does not target a specific cell. No immune response + not toxic + more suited for ex-vivo therapy (outside the body). Advantage: Does not trigger an immune response Can carry large genes Disadvantages: Less efficient than viruses at entering the cell and therefore at delivering the genes to the intended cells. In- Vivo(direct):The Vector can be injected or given by IV to a specific tissue in the body and infected by the individual cells. (learn.genetics) Ex-vivo (cell-based): This is when the cells are taken from the patient and the vectors are used outside the body to deliver the genes to the cells then grown in culture. After the cells are tested to make sure that they are working properly and have been altered they are put back in the targeted area of the patient. No virus given to patient =Less likely to trigger an immune

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