Engineering And Genetic Modification

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Engineering and Genetic Modification

Introduction

In recent years, our knowledge of the genetic and molecular bases of human diseases is rapidly improving, that is, what genes and gene chains take part in processes such as embryonic development, growth, aging and their functioning in organs or organs orIn high incidence diseases. 

In this section, it will be explained about the introduction of healthy genes in sick cells;In addition, it will be developed as neurodegenerative diseases can be treated through gene therapy. In the first place, the introduction of healthy genes can be given in sick cells;In this strategy, what is sought is to correct the gene altered by the correct gene. 

To do this, in theory, recombination procedures can be proceeded that allow to exchange the gene mutated by the correct gene, to optimize the introduction of DNA in the cells, transfer vehicles are required, also called vectors, they are the transporters of the sequences ofhealthy genes. At present, there are viral and non -viral vectors. 

Among the viral vectors, especially those derived from retrovirus and lentivirus, adenovirus and adenoasociated viruses are especially highlighted. The most relevant non -viral methods from the point of view of their potential clinical application are, the use of liposomes of various compositions, molecular conjugates and direct purified DNA transfer. To introduce a gene into a cell the vectors mentioned above are used and these are those that normally transport genetic material to cells, such as viruses. 

Developing

Genetic therapy in cancer treatment;Traditionally the approaches involved the destruction of cancer cells with chemotherapeutic agents, radiation or surgery. Gene therapy has obtained satisfactory results such as the reduction of large solid tumors. Most medical studies for the application of gene therapy are directed to cancer disease, being the main methods used.

 The increase in the antitumor cell immune response that consists in educating the cells of the immune system that have been removed from the patient to adopt characteristics that allow them to exterminate cancer cells when they are finally reinjected to it;We also have the introduction of drug activating genes inside the tumor cells that consist of the selective introduction of genes that encode and are susceptible to drugs that lead to the production of enzymes that convert produce cytotoxic metabolites that disintegrate tumor cellswho have reproduced.

 We also have the use of bone marrow cells, which is generally used in hematological disorders therapy, and consists of transmitting to the hematopoietic progenitor cells Chemio protection genes, which enable the highest dose administration of chemotherapeutics;Finally we have the use of ribzymes and antisntido or ‘antisense’ technology. The ribzymes are RNA with catalytic activity that work by increasing the degradation of newly translated RNA, reducing unwanted specific proteins, a factor that is sometimes linked to tumor alterations. 

Antiscentide technology refers to OLIGONUCLEOTIDES of RNA that have no catalytic activity, but are complementary to a gene sequence. These oligonucleotides could act by different mechanisms such as: the blocking of the processing of the transcribed RNA of the DNA, impediment of the transport of the RNA-Antisense complex to the cytoplasm and blocking of the beginning of the translation. On the other hand we have a phase I clinical study of a group of people with malignant melanoma, in which melanoma cells with interferon-gamma gene, cryoprevied and inactivated to be used in the form of vaccine. 

Increasing cell doses were supplied once every two weeks to 13 patients to evaluate their immune response and the benefits of the same. Eight of them pointed out some type of immune response. In 2 patients who obtained a significant immune response there was tumor regression, and another 2, with a lower response, showed transitory nodule resolution. Second, through genetic therapy, neurodegenerative disease treatments can be performed. 

This is because most of the disorders that are presented at the level of brain tissue are manifested with neuronal loss;Hence, an output to achieve the restoration of damaged tissue is the implant of cells capable of differentiating neurons, the release of genes with neuroprotective and neurostauradora or enzyme function whose function is affected as a result of neuronal loss. In this sense, two clinical trials of gene and ex vivo gene therapy have been carried out. 

conclusion

The first consisted of the release of the enzyme Glutamic Acid Discarboxylase, responsible for the synthesis of γ-amino-butyric acid to relieve the characteristic symptoms of Parkinson’s disease. A month after surgery no additional fever or neurological dysfunctions had been detected. Recently, phase I of a study was carried out in which genetically modified fibroblasts were implanted to release NGF in eight patients with Alzheimer’s disease. 

After 22 months of monitoring, no adverse effects associated with the procedure were detected, the cognitive function of six patients improved and in an autopsy a significant response to the release of NGF was demonstrated. As a last example of neurodegenerative diseases in which various approximations of gene therapy have been carried out, we will quote ataxias, in which a virus capable of reaching the medullary dorsal nodes, one of the most affected areas inFriedreich’s ataxia. 

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