“Gene therapy” and “Gene doping” is an extension of the recombinant DNA technolo
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Question
“Gene therapy” and “Gene doping” is an extension of the recombinant DNA technology used for gene cloning we are discussing in class. While gene therapy generally implies that the genetic modification of the individual is used for therapeutic purposes, gene doping implies making genetic modifications to improve humans, not necessarily to cure a human disease. This link provides some good background (http://www.genome.gov/10004767 (Links to an external site.) ) while this link (http://www.nature.com/scitable/topicpage/sports-gene-doping-and-wada-764 (Links to an external site.)) is a good introduction to this controversy. For this week’s forum I would like you to research this topic and consider the pros and cons of gene therapy. Do your own research and find examples of how gene therapy is currently being developed or used. Do the benefits of this technology outweigh the potential dangers? Are the potential uses of this technology such as gene doping acceptable? Are there other potential uses of this technology that would not be acceptable, genetic modification of human embryos (designer babies)? As society how should this issue be discussed and who should be responsible for regulating it? For your post make sure that you take a stand on this topic one way the other and support it with outside research.
Explanation / Answer
Gene therapy is an experimental approach to treat the disease by inserting therapeutic genes or changing gene expression levels in the patient’s cells. Genes are nucleic acid polymers; these therapeutic genes used in gene therapy generally encode a functional gene that replaces mutated gene. Potential treatment for single gene genetic disorders such as haemophilia, muscular dystrophy, cystic fibrosis, etc. was demonstrated. Treatment of cancer, AIDS (acquired immune deficiency syndrome), Alzheimer’s disease and rheumatoid arthritis are also under active investigation. Approaches of gene therapy include the following.
Two types of gene therapy are somatic gene therapy and germ line gene therapy.
Somatic gene therapy: In this method, the genome is introduced into somatic cells of the patient. So, the changes that occur after therapy will remain with the patient only and cannot be inherited.
Germ line gene therapy: The normal genome is introduced into the reproductive cells of the patient. So, the consequences of the therapy can be inherited.
Viral vectors are being used as carriers of normal genes that replace defected genes within the cells. The success of gene therapy is partly depending on the integration of new gene into the host genome, which in turn depends upon the retroviral integration system. Non-viral vectors are also developed in the recent past. To deliver the target genes, two techniques are being used, namely ex-vivo and in-vivo methods.
Pros of gene therapy: Gene therapy is a very suitable therapy for genetic diseases caused due to defects in known single gene disorders. This may offer cure for some diseases. With the help of reproductive therapy, carrier genes can be deleted and thus, we can prevent the passage of defective genes to the offspring.
Cons of gene therapy: Identification of defective genes using DNA probe and availability of normal genes is pre-requisite to gene therapy. It is difficult to treat diseases controlled by multi genes. Cost of therapy is high, which may not be affordable by most of the patients. Safety issues has no clarity in gene therapy, there is gap in understanding.
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