Human Gene Therapy

Human gene therapy Each of us has about 6 defective genes. Most of us are not harmfully affected by the gene we are missing. We have two copies, one from the mother and one from the father. In most cases, because its potentially harmful gene is recessive, its normal counterpart performs all tasks assigned to both. Only when we inherit two copies of the same recessive gene from parents we will develop the disease. Every year, quite a few children are born with genetic diseases. It happens because each of our parents suffers from the same genetic defect (Rigby 1995).

The first approach to human gene therapy began in 1990. Gene therapy involves treating people suffering from disease by placing good genes appropriate for their system so that they can fight disease. This research focuses only on situations in which certain genes are missing in the human body or genes are not functioning properly. The gene is added by inserting DNA into the patient's cell. The success of gene therapy is limited by many factors. For example, a subject that is typically receiving treatment has multiple problems that may interact in some unknown way and that person's immune system may be for a new gene introduced There is. Since 1990, gene therapy has made little progress.

Gene therapy is the basis of the movie "Am Am ​​Legend" to change humanity and the TV program "Will Gene Therapy". In 1994, gene therapy was a plot element of The Erlenmeyer Flask which was the first season of the X file. It is also used as a gateway to stars as a means to enable humans to use ancient techniques.

Gene therapy uses DNA as a drug for treating diseases. Gene therapy was originally conceptualized in 1972 and the authors urged attention before starting gene research on humans. The first FDA approved gene therapy trial in the US was held in 1990. This is a 4 year old girl named Ashanti DeSilva who received ADA - SCID treatment. This is a disease that prevents her from fighting infection. Dr. W French Anderson is the principal investigator of clinical trials and works at the National Heart, Lung and Blood Laboratory. Since then, more than 1,700 clinical trials have been performed using a variety of gene therapy technologies.

Gene therapy has now become a mature field since the first application of human gene transfer over more than 20 years and has gradually overcome some obstacles that hinder clinical success in the early stages of application. To date, most gene therapy clinical trials have used viral vectors as very efficient nucleic acid delivery vehicles in vivo and ex vivo. Here we summarize the current state of clinical application of viral gene transfer, with particular emphasis on molecular characterization of major classes of viral vectors and information obtained from clinical trials of gene therapy to date.