Genes, which are carried on chromosomes, are the basic physical and functional units of heredity.

Genes are specific sequences of bases that encode instructions on how to make proteins. When genes are altered so that the encoded proteins are unable to carry out their normal functions, genetic disorders can result. Gene therapy is a technique for correcting defective genes responsible for disease development. Currently, the most common vector is a virus that has been genetically altered to carry normal human DNA. Scientists have tried to take advantage of this capability and manipulate the virus genome to remove diseasecausing genes and insert therapeutic genes. The Food and Drug Administration (FDA) has not yet approved any human gene therapy product for sale. Current gene therapy is experimental and has not proven very successful in clinical trials. In spite of continued great difficulties with technical implementation, initial successes in somatic gene therapy are on the horizon, for example in treating adenosine deaminase deficiency and chronic granulomatous disease. There are also initial indicators that point to the efficacy of gene therapy in treating chronic lymphatic leukaemia and haemophilia B. But successes in clinical testing come with their share of setbacks, such as the currently slight margin of therapeutic effectiveness over unwanted side effects. This underscores the necessity to carefully assess risks and benefits and also shows that there is still a dire need for more research on gene therapy. Basic research should be conducted in direct collaboration across disciplines, using animal models and clinical studies. An acute need for research exists, providing a big horizon for today's scientists to combat over the vectors for a remedy.

What is a gene therapy?
• Genes, which are carried on chromosomes, are the basic units of heredity. • It consists of specific sequences of bases that encode instructions on how to make proteins. • When the encoded proteins are altered it creates a disorder, which is the genetic disorder and the gene becomes defective. • This gives the scope for disease development. • For correcting defective genes responsible for disease development a technique called Gene therapy is used.

Gene therapy uses a modified virus or other carrier to transport genetic material into cells that have defective or missing DNA. Currently, the most common vector is a virus that has been genetically altered to carry normal human DNA. Scientists have tried to take advantage of this capability and manipulate the virus genome to remove diseasecausing genes and insert therapeutic genes. For example, it is being studied as a way to produce insulin and to treat complications such as diabetic neuropathy, foot ulcers and high blood pressure. This technology may have wide-ranging implications for the treatment of diabetes.

•The endocrine system is made up of the glands and hormones that regulates the physical and metabolic activities of the body. •A disorder in the body’s ability to break down blood sugar is called as DIABETES. • It is of two types:2. Type-1 and 3. Type-2 diabetes. • In type 1 diabetes, the body produces no insulin.

• It is also called as juvenile diabetes. • In type 2 diabetes, the body does not produce enough or cannot use insulin. •It was once called adult diabetes because it is usually diagnosed in adulthood.

Role of gene therapy in treating diabetes.
• Clinical trials are assessing the effectiveness of gene therapy to treat diabetic ulcers. • Scientists have developed a topical application of a gene that is designed to stimulate repair cells in and around the wound. • In diabetic patients at risk for foot or amputation, on treatment of gene therapy reported resolution of chronic pain and wound healing. • Much of the research in diabetic gene therapy has focused on the stimulation of insulin and the insulin-producing beta cells of the pancreas. leg

• Laboratory researchers are exploring gene therapy to extend the life of human beta cells. • Lower glucose (blood sugar) by delivering insulin genes to the pancreas. • Extend the life of pancreatic islet cell transplants and prevent problems such as tumors. • It is also be used to treat complications ranging from foot ulcers to kidney disease.

• The goal of gene therapy research is to prevent, treat, relieve the symptoms of or cure diabetes.

• Researchers have also used gene therapy in the laboratory to: • Prevent the onset of hyperglycemic in normal-weight diabetes-prone mice, a potential advance in preventing type 1 diabetes. • Increase beta cells and lower blood glucose levels in mice with a condition similar to type 2 diabetes.

• Restore the growth of rodent nerves and blood vessels that were damaged diabetes.

Benefits and risks.
• The potential benefits are great. • Gene therapy in the future may be able to control the production of insulin, thus freeing patients from the need for injections, pumps and other methods of insulin administration. • One of the major variations in gene therapy is the choice of a vector. •Vectors, the means of delivering gene therapy to a cell are harmless. •In a few cases, however, viral vectors have been involved in causing disease or sparking a reaction in people during trials of gene therapy, sometimes fatally.

Ongoing research on gene therapy.
• The research in gene therapy is still in early stages and scientists are exploring many potential uses to treat diabetes and other conditions. •Stem cells may be used to create insulin-producing beta cells or islet cells. • Scientists are researching the possibility of using DNA-based vaccines to increase the immune system’s tolerance. • Researchers are developing cellular depots to store insulin and secrete it in response to an increase in glucose.

• Researchers have reported preliminary success in using gene therapy to treat diabetic rodents. • Extensive testing is required before any studies completed on animals can be undertaken on human subjects. • Gene therapy is a growing field of research that will likely have many advances and setbacks before a cure for diabetes may be found. • In the meantime, it is important that patients control their diabetes by exercising, eating healthily, performing glucose monitoring, taking medication and following the other steps in a physician’s treatment plan.

Sign up to vote on this title
UsefulNot useful