Abstract
Genes are regions on chromosomes which carry specific information for the production of proteins. Advancement in science, especially in medical research, has led to the identification and isolation of genes responsible for human diseases. A person carrying the defect in a specific gene might develop disease, which may be inheritable. In gene therapy, nucleic acid is the therapeutic agent. In this, the defective genes responsible for the disease are genetically modified in patients in order to achieve a therapeutic goal and return the individual to good health. The therapy is achieved by using a viral vector or gene delivery vehicles. The steps involved in the therapy in which the gene of interest enters the target cell via viral vector and begins expression are referred to as transduction. Gene therapy can be used to treat diseases caused by mutations in the patient’s own DNA (inherited disorders, cancers) as well as infectious diseases and is particularly valuable in cases where no conventional treatment exists or where that treatment is inherently risky.
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Some Selected Resources
http://www.fda.gov/biologicsbloodvaccines/cellulargenetherapyproducts
http://www.fda.gov/biologicsbloodvaccines/cellulargenetherapyproducts
National Cancer Institute Factsheet
Some text directly from National Cancer Institute Factsheet as source
Successful gene therapy trial in Parkinson’s Disease
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Gupta, V., Sengupta, M., Prakash, J., Tripathy, B.C. (2017). Gene Therapy. In: Basic and Applied Aspects of Biotechnology. Springer, Singapore. https://doi.org/10.1007/978-981-10-0875-7_17
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