Gene Therapy

1207 Words 5 Pages
Despite early failures that dismissed gene therapy as a viable means to treat disease and genetic disorder, decades of research have resulted in the development of safe and effective vectors, methods of targeting particular types of cells, and techniques to minimize and manage immune responses. In the process, scientist now know more about the disease-causing genes themselves than ever before, paving the way for the development of successful gene therapy treatments. As of now, people have been successfully treated for disorders and diseases including hereditary blindness, immune deficiencies, and even cancer. Inherited immune deficiencies, such as Severe Combined Immune Deficiency (SCID) and ADA deficiency, were one of the first genetic disorders …show more content…
Some speculative uses for gene therapy in the future include fertility applications, improving athletic performance, and human genetic engineering. The gene therapy method known as CRISPR interference, which can be used to alter the germline (cells that may pass their genetic material to future generations) of organisms and humans, and modifying the genes of food crops, may be used with specific endonuclease enzymes for genome editing and gene regulation. By delivering the CRSIPR associated Cas9 protein along with appropriate guide RNAs into a cell, the organism’s genome can be cut or modified at any desired location. Successful experimentation on mice has demonstrated that this method can be used to restore fertility by transplanting spermatogenical stem cells from another organism into the testes of a mouse, restoring spermatogenesis and fertility. Genetic engineering in this way can be used to change physical appearance, improve metabolism, improve physical capabilities and mental faculties, such as intelligence, and even extending human lifespan. Theoretically, since all diseases, whether caused by genetic mutations or environmental factors have a genetic origin, the use of gene therapy, if perfected, can essentially cure everything, even death. However, before genetic engineering can take place in this way, gene therapy must overcome many unsolved issues. For one, integrating DNA into patients’ genome is currently inefficient and the rapidly dividing nature of cells prevent treatments from achieving long-term benefits. Another problem is that the body tends to reject foreign genetic material causing immune responses that may reduce the effectiveness of treatments. Also, successive treatments become less effective since the immune system becomes adept at fighting viruses that have been introduced

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