Gene therapy is an experimental medical treatment that uses specific types of genes to treat or prevent several types of diseases. Gene therapy is also used to treat several hereditary diseases, wherein the mutated defective gene is replaced with the functional gene. Gene therapy is being widely studied by researchers all over the world for the treatment of several diseases such as immune deficiencies, hemophilia, Parkinson’s disease, cancer, and even HIV, through different approaches. Three primary approaches that are being studied and practiced in the gene therapy are replacement of the mutated disease-causing gene with the healthy gene, inactivation of the mutated gene, and introduction of the new gene to fight against the disease. In the gene therapy treatment, a functional gene is inserted into the genome of an individual’s cells and tissues by using a carrier known as ‘vector’. Viruses is the most common type of vectors used in gene therapy, which is genetically altered to carry the normal human DNA. Over the last few years, gene therapy has emerged as a promising treatment option for several diseases including inherited disorders and certain types of cancers and viral infections. However, the treatment is still reviewed for its effectiveness and safety. Currently, the gene therapy is being tested only for those diseases for which no other treatment is available.
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The demand for gene therapy is greatly influenced by the increasing number of patients with cancer, hereditary disorders, and viral infections. The International Agency for Research on Cancer has estimated that more than 21.7 million cases of cancer are likely to be diagnosed worldwide and more than 13 million deaths would occur due to cancer across the world by the end of 2030. Moreover, according to Joint United Nations Programme on HIV and AIDS () in 2015, there were approximately 36.7 million people living with HIV/AIDS across the globe. Technological advancements and several pre-clinical and clinical trials for the treatment of different disease are likely to boost the gene therapy market during the forecast period. Also, successful clinical trials of gene therapy for the treatment of central nervous system disorders would open new avenues for the global gene therapy market in the near future. Moreover, increase in R&D budgets by commercial players and governments for the development of safe and effective gene therapy for various diseases would propel the global gene therapy market in the near future. However, high cost of gene therapy treatment and unwanted immune response in some cases are some of the restraining factors for the market.
The global gene therapy market can be broadly segmented based on type of gene therapy, type of vector used for gene transfer, disease indication, and geography. Basically, there exist two types of gene therapy: germline gene therapy (which involves the modification of genes inside the germ cells i.e. sperms and ova) and somatic gene therapy (where the therapeutic gene is inserted into the body cells or tissues and not in germ cells or gametes). The somatic gene therapy is widely studied and practiced for the treatment of cancer and infectious diseases. Use of the germline gene therapy for the treatment of hereditary disorders is prohibited in countries such as Germany, Switzerland, and Australia, due to and long-term effects on the future generation. Based on type of vector used, the market has been segmented into viral vectors and non-viral vectors such as bacteria and plasmid DNA. Viral vectors are most commonly used and studied. The viral vectors segment accounts for a major share of the global gene therapy market. Based on indication, the global gene therapy market has been segmented into infectious diseases, genetic diseases, cancer, and other diseases.