Gene Therapy Saves Lives

Gene therapy saves life Tim was diagnosed as a rare and fatal illness this morning. He is only five years old and he is in front of him for the rest of his life. He accepts the disease, even his parents are not his fault. This disease occurs by anyone who has a defective gene. Last year, he was afraid that he might catch any common insects and was afraid that they might die, so the disease allowed Tim to live in a sterile foam. He has severe joint immunodeficiency or SCID. In this disease, genes involved in the body's immune system, called adenosine deaminase, are missing.

LUXTURNA is a gene therapy treatment that can save a day and is also a big medical advancement. LUXTURNA formally approved by the FDA Jury Committee will be the first gene therapy and remedy for hereditary disorders in the United States. LUXTURNA is a novel gene therapy developed by the pharmaceutical company Spark Therapeutics. Treatment involves injecting a patient with genetically modified adenovirus carrying the gene to counteract the loss of RPE 65 protein. The purpose of this treatment is to at least partially restore the function of the visual cycle and help resolve visual impairment. The results of clinical trials show that treated individuals show clinically meaningful improvement in visual perception and function.

Many genetic diseases that may be treated by gene therapy are extremely rare, some of which affect only 1 in 1 million. Although gene therapy can save the lives of these patients, the high cost of developing therapy makes it unlikely for it to be appealing to pharmaceutical companies. It is very expensive to develop new treatments, including clinical trials that government approval requires. Because a limited number of patients can recover these costs, developers may not be able to make money by treating this rare genetic disorder. Some patients may never afford to buy