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What is a genetic disorder?
Genetic disorders are the defects in the genes of a person. Most of them are multi-factorial hereditary illnesses, like Huntington disease and Parkinson’s disease. A few of them occur due to environmental hazards. It is estimated that there are 4000 to 6000 genetic disorders, which have been diagnosed already (Genetic Disorders, 2014).
Interest and relevance
Genetic disorders will hurt the patients, and that makes their life horrible. Though there are no treatments for these rare genetic conditions, supportive care can bring relief to some extent. However, it is found that most of such treatment strategies fail to change the mutation of the genes. It is in this context that the application of gene therapy has increased the hope of medical professionals in overcoming and controlling such failures in the treatment of genetic disorders (How are genetic conditions treated or managed? 2014).
Gene therapy and principles of heredity
Gene therapy is a treatment used for changing gene functions. Acquiring knowledge about molecular and genetic aspects of the disorders is the guiding principle behind it. It is found that the human genome consists of 25,000 genes and that the identified 18000 mutations are responsible for the hereditary disorders. Therefore, knowledge about the relationships between the genotype and phenotype can be applied for the treatment of these disorders.
Application of molecular genetics for genetic variation
Since the inherent metabolism errors caused by the shifts in genes influence the construction of certain enzymes, dietary deviation and enzyme substitution treatments can bring progress in some genetic disorders. However, the improvement will be very slow. Nevertheless, Gene therapy is a different technique, which uses DNA as a medicine to bring changes in the cells of a body for correcting the defective genes. The normal genes are produced through PCR from the DNA of another person, and the therapeutic DNA derived is injected into the patients by means of vectors. These vectors install a copy of the mutated gene to replace the protein, responsible for the disease (How are genetic conditions treated or managed? 2014).
Genetic disorders cured with gene therapy
Some of the genetic disorders that can be treated with gene therapy are Severe Combined Immune Deficiency and Chronic Granulomatus Disorder. For improving the immune system of the patient, the bone marrow is transplanted after correcting the cells by inserting ADA, as a treatment for these two disorders. Other major genetic disorders like hemophilia and congenital blindness are curable through gene therapy. Acquired diseases are treated with different gene therapies. Such therapeutic strategies include gene vaccines and oncolytic virotherapy. However, for neurodegenerative illnesses like Parkinson’s, the best therapy is the Novel gene. Different acquired diseases are also treated with gene therapy techniques. Research is in progress to develop new gene therapy techniques to fight viral infections, diabetes, and heart diseases (What Diseases Can Be Treated with Gene Therapy, 2011). The recent discovery of chromatin architecture that controls the gene activity is a milestone in the diagnosis of genetic disorders (Institut de Recherches cliniques de Montreal, 2014).
Bioethical issues
Despite all these developments in gene therapies to treat genetic disorders, applying the gene therapy itself is a challenge for medical professionals. The main ethical issue is how one could differentiate good and bad gene therapies. What happens if people change their basic human traits to master others? Moreover, the insertion of new genes into the cells of a body is an elaborate and costly process, and developing gene therapy for rare genetic disorders is very sophisticated. It needs many clinical trials to obtain government approval, prior to its application as specific gene therapy. These are long-standing issues in gene therapy treatment for genetic diseases as well as acquired diseases (Genetic Science Learning Center, 2014).
References
Genetic Science Learning Center (2014) Challenges in Gene Therapy?.Learn.Genetics. Web.
Genetic Disorders. (2014). Web.
How are genetic conditions treated or managed?. (2014). Web.
Institut de recherches cliniques de Montreal. (2014). Important discovery for diagnosis of genetic diseases. ScienceDaily. Web.
What Diseases Can be Treated with Gene Therapy. (2011). Web.
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