Gene Therapy and its Novel Approach

Gene therapy could enable patients to live without the need for ongoing treatments or the burden of daily disease management. We're going beyond helping people manage their diseases. We want them to thrive in every stage of life.Gene therapies are being used to treat a small number of diseases, including an eye disorder called Leber congenital amaurosis and a muscle disorder called spinal muscular atrophy. Many more gene therapies are undergoing research to make sure that they will be safe and effective. Genetic tests may be used to confirm a diagnosis in a symptomatic individual or used to monitor prognosis of a disease or response to treatment. Predictive or predispositional genetic testing can identify individuals at risk of getting a disease prior to the onset of symptoms. The idea that a gene can be delivered into specific cell types and its expression can lead to therapeutic efficacy, dramatically improving the patients' quality of life, was originally introduced by Theodore Friedmann 45 years ago and was later strongly encouraged and realized by George Stamatoyannopoulos, one of the founding members of the American Society of Gene and Cell Therapy (ASGCT).It has been over 30 years since visionary scientists came up with the term “Gene Therapy,” suggesting that for certain indications, mostly monogenic diseases, substitution of the missing or mutated gene with the normal allele via gene addition could provide long-lasting therapeutic effect to the affected patients and consequently improve their quality of life. This notion has recently become a reality for certain diseases such as hemoglobinopathies and immunodeficiencies and other monogenic diseases.