The Pros And Cons Of Gene Therapy

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Gene therapy methods

Gene therapy is one of the most rapidly growing techniques in the medical field. One out of ten people are affected by genetic disorders. Defective genes that code for an incorrectly formed protein, resulting in a severely hindered function, cause genetic disorders or process that are usually lethal. The essential idea was to replace the defective genes causing the disorder by introducing a confirmed healthy form into the patient through some sort of vector. Vectors are fragmented down into two groups, viral and non-viral. Viral vectors have been prosperous in some gene therapy applications, but there are possibilities that the patient’s immune system may defend against the virus and render the application insufficient. These vectors have patient’s immune system defend against the virus and render the application unusable. It also, has been successful in treating patients with severe combined immunodeficiency (SCID), in part due to the weakened immune system of the patients. Methods that are used in the following papers utilized viral vectors to introduce the desired genes into the patients. Severe combined immunodeficiency that is linked to the …show more content…

have developed a new viral vector to deliver the cystic fibrosis. PIV vectors was made through the use of recombinant human PIV3 encoding GFP, CFTR, and F508CFTR cDNA as separate genes generated from the cDNA antigenome of full-length hPIV3 JS strain. After retrieval, PIV replicated in HEp2 cells to a titer comparable to the JS wild-type strain suggesting that GFP, CFTR, and F508CFTR did not adversely affect growth capacity of PIV in producer epithelial cell lines (Zhang et al., 2009). In vitro model of human airway epithelium was used to test the effectiveness of parainfluenza virus with the CFTR gene (Zhang et al., 2009). There was a success in delivery of the CFTR gene to the surface cells (Zhang et al.,

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