Gene therapy

Hereditary diseases bother people and lead to death. However, these unfortunate people are now expected by new information and research, what is called gene therapy. Gene therapy is a technique used to correct defective genes that cause disease progression. It has been circling for a while and is getting more advanced with the times. The experiment is an ongoing process of gene therapy. Ethical problems are programs associated with the use of programs.

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.

The first clinical trial of gene therapy was done in 1990 by the National Institutes of Health in Washington, DC, and it was done dozens of times thereafter. Until this year, the first gene therapy began at the clinical trial stage and received the first gene therapy approved by the Federal Drug Administration (FDA). Novartis' Kimuria (tisagenlecleucel) approved on August 30, 2017 is a remedy for children and young patients with certain acute lymphoblastic leukemia (ALL). It is T cell immunotherapy, which means that each dose is a patient's own T cell, a customized treatment made using immune cells found in the blood.

Current gene therapy research focuses on treating individuals by targeting somatic cells such as bone marrow or blood cells. This gene therapy is not handed down to a human child. However, gene therapy can target eggs and sperm cells (reproductive cells), which makes it possible to transfer the inserted genes to future generations. This method is called germline gene therapy. The idea of ​​germline gene therapy is controversial. It can protect future generations of families from specific genetic diseases, but it may have unexpected long term side effects, which may affect fetal development in unexpected ways. People affected by germline gene therapy have not yet been born, so we can not choose whether to receive treatment. Because of these ethical concerns, the US government will not allow the Federal Fund for human germline gene therapy research.