Gene Therapy In Genetic Engineering Applications

Gene Therapy In Genetic Engineering ApplicationsGene therapy is one of the applications of the transmittable engineering. It is a technique which correcting defective agent that is responsible for ailment training (cited Safdar, 2010). Gene therapy also involves the addition of healthy and useful copy of the faulty gene into the target prison cells of the body. Gene therapy generally works by inserting a sane gene into the genome to replace an abnormal gene in the target cells. There are two types of gene therapy. Firstly, germ line gene therapy which involves modification of patrimonial material in the gamete cells that would be heritable and can be pass through to the next generation. abetly, embodied gene therapy which involves insertion of alterative gene into somatic cells of the patient that only effect to the individual patient only and exit not be inherited by patients offspring.( cited in Ten Tailed Fox, 2009). sundry(a) genetic disorder complaints commonly are ca apply by faulty genes. With gene therapy, instead of treating the symptoms of the disease, treatment also target and correct the underlying cause of disease and eventually improve the faulty genes to normal genes. One of the genetic diseases that apply gene therapy as resource treatment is Cystic Fibrosis.Cystic Fibrosis ( cf is one of the close to well-known hereditary lethal diseases. A study conducted by UK Cystic fibrosis Gene Therapy Consortium (UK CFGTC) stated that approximately, 50 000 people and 1 in 2500 red-hotborns somewhat the world are touch. CF is an autosomal recessive disorder which caused by the mutation of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein that leads to abnormally thick secretion of mucus. The organs most affected by CF are the lungs, gut, pancreases, liver and reproductive tract, but lung infection is the most serious affected by CF. (cited in Metharom, 1997). uninterrupted inflammation, damaged lungs and respiratory fail ure are results of repeated cycles of infection that may leads to death.With the development of effective treatment to overcome CF, gene therapy was introduced by scientist in 1990 which promises new opportunity for CF patients to train a better and long life span. This gene therapy involves replacing the detective CFTR gene with the normal one in the affected cells. As the results, the functional CFTR are being produced in all target cells and the CFTR gene volition then conducts the production of normal CFTR protein which acts as a sensitive that allows releasing chloride and other ions. But this is opposite with people who have CF, where CFTR protein is detective and the cells do not discharge chloride ions.Although, gene therapy has the capability in treating CF, it still faces with several(a) issues. This report consists of history of CF gene therapy and genetic techniques of gene therapy on CF. Research findings also include the advantages and disadvantages of using gene t herapy in treating CF.2.0 BACKGROUND GENETICGene therapy has shows that much positive feedback in treating genetic diseases although there are many failures happened. Gene therapy for CF first discovered in 1990 when scientist treated defective CFTR successfully and they added the normal copies of the gene to laboratory cell structures. The first experimental CF gene therapy treatment was given to CFs patient in 1993 (cited in Schoenstadt, 2009). The table below shows the major events that occurred due to development of gene therapy in treating Cystic Fibrosis.3.0 GENETIC TECHNIQUESIn gene therapies, there are terce main techniques that are being used in treating diseases. The first is Gene addition, in which the genetic material is added to the target cells with no attempt to incorporate it into chromosomes. Second one is techniques of gene repair that replace abnormal segments of DNA in defective genes in their normal chromosomal site. The last one is Gene exchange techniques wh ich allow deletion of the abnormal gene from its chromosome and replacement with a normal gene. (cited in Sade RM, Khushf G., 1998).In order to treat CF, the common technique used is Gene replacement by using vector. Vector is a DNA molecule (virus) act as a medium to transfer foreign genetic material into another cell. Instead of introducing new genetic material into target cells vectors are also agents to which new genetic material is attached. Diagram below shows the functional of a virus acts a vector in gene therapy.First step to do is designing a vector by modifying the gene of the virus. We learn to remove the rep gene which codes for proteins that responsible for viral replication as well as cap gene, which codes for capsid structural proteins. Then, we insert the normal CFTR gene in the virus. Second step is producing the virus. After inserting the CFTR gene in the virus, we transfer the vector into a packaging cell line so that the vector will combine with protein needed to assemble the virus. Next, we collect the virus sample from the growth medium and add into the Petri dish containing the CF cells. The virus give effect to the cell as it integrates the DNA into specific location on chromosome 19. Last step, we transfer the vector into CF patient. CFTR gene in the cell will transcribe into mRNA and this induces the cell to produce normal CFTR protein in the target cells. Diagram below show the mechanism of vector in treating Cystic Fibrosis.4.0 SOCIAL ISSUESIn new years, many types of techniques have been developed to identify genes associatedwith specific diseases. Society always debate over the ethics of using gene technology to treat kind-hearted beings when recombinant DNA research began. Tables below show advantages, disadvantages, ethical and social of practicing gene therapy to treat genetic diseases such as Cystic Fibrosis.ADVANTAGES OF GENE THERAPYGene therapy promotes in providing better and normal life for those who practise the the rapy. (Ten Tailed Fox, 2009).The practice of gene therapy on CF patients can solve their problems by replacing none functional gene with functional one. (cited in S. Muniba, 2010)Gene therapy has high success rate compared to other methods in treating CF disease.Gene therapy helps patients to avoid from practicing unnecessary treatment and alternative which may be dangerous to them.SOURCE http//www.biotecharticles.com/Genetics-Article/Gene-Therapy-Advantages-and- Disadvantages-271.htmlDISADVANTAGES OF GENE THERAPYScientist stated that correcting the defect in people who had diseases is much harderthan achieving correction in cells in the laboratory .It operator that there are also numbers of trial that results in failure. (cited in Healingwell.com )Practice of gene therapy using vector may induce immune reaction in the patients. The gene that is injected or sprayed into patients target cells may trigger antibodies to kill the vectors. ( cited in William, 2000).This is because virus is used as a vector to transport the therapeutic gene.The use of gene therapy in treating CF will cause in diminishing the gene pool. Their patient s next generation will have to survive with the changes in their genomes.(cited in Sade RM, Khushf G., 1998 ).Chance of inducing a tumor .If the DNA is integrated in the wrong place in the genome, for utilisation in a tumor suppressor gene, it could induce a tumor. (cited in Healingwell.com)SOURCE http//academicdepartments.musc.edu/humanvalues/pdf/gene-therapy.pdfIn conclusion, gene therapy is a best solution in treating cystic fibrosis because it has the high chances to eliminate this genetic disorder disease. Muniba stated that gene therapy could be last cure for every genetic disease. People who take gene therapy will make up their mind if they also face with this problem because this type of treatment can be the last hope for them in treating hereditary disease like Cystic Fibrosis.Government should concerns regarding the harms tha t may be associated with gene therapy and the beneficial uses of gene technology are required.(1547 words)