However, it does have various treatments. However, researchers recently found that a mutation in the DMD genes – an in charge of proteins necessary for muscular contractions  –is mostly responsible for Duchenne’s muscular dystrophy. She is interested in robotics and has mentored a robotics team. Your email address will not be published. Chronic Granulomatus Disorder (CGD) CGD is a genetic disease in the immune system that leads to the patients' inability to fight off bacterial and fungal infections that can be fatal. The first patient was operated upon in early 2007. Severe Combined Immune Deficiency (ADA-SCID) ADA-SCID is also known as the bubble boy disease. There are a couple of ways to perform this procedure but most of them are in the infancy phase. Using similar technologies as in the ADA-SCID trial, investigators in Germany treated two patients with this disease, whose reconstituted immune systems have since been able to provide them with full protection against microbial infections for at least two years. She is interested in robotics and has mentored a robotics team. Although some types of gene therapy are aimed at specific cancers, early studies show promise for mesothelioma treatment. She was raised in California and recently moved to Pakistan. She is currently studying her ‘O’ levels in Lahore. About Author / Additional Info: There is a whole world of the unknown still waiting to be explored by gene therapy. Finding a reliable way to get genetic material into cells 2. More clinical trial information. The possibilities are endless in this new and exciting field of study. Additionally, she has taken part in many robotics competitions, both international and local, Your email address will not be published. Among the most notable advancements in gene therapy are the following. Additional eligibility requirements must also be met, but these will vary depending on the individual study. In a clinical trial conducted in the United States, the therapeutic gene was introduced into the liver of patients, who then acquired the ability to have normal blood clotting time. However, these are just a few examples of the possibilities that ‘gene therapy’ offers. Neurodegenerative and other acquired disease can also be treated with gene therapy and give healthy results. The main aim of gene therapy is to cure a disease by providing the patient with a correct copy of the defective gene. 1. Gene therapy is a technique to replace the faulty or missing gene with a healthy copy of the same gene. The inspiration for this was derived from the natural defense mechanisms of bacteria and archaea to fend off attacks from viruses and other foreign bodies. For example in the cancer disease, patients are treated with multiple genes and therapeutic gene vaccines. As a result, the KamiCas9 invented, a type of CRISPR-Cas9. To help understand gene therapy, here is a general example of a gene therapy and how it moves from research to a potential therapeutic reality: Scientists discover the genetic basis of a disease, which might be a mutation in a single gene 8; Scientists target their research on ways to modify the specific mutation 8 Troxel inherited the faulty genes through both parents. Bibhu Dev Misra is an independent research investigator and writer on anc... Saadeqa Khan is a freelance science journalist based in Quetta. Blindness Leber's congenital amaurosis (LCA) is a rare inherited eye disease that appears at birth or in the first few months of life, and affects around 1 in 80,000 of the population. Gene therapy is a medical therapeutic process involving the delivery of the gene into cells to treat diseases. Scientists work to target cancer through gene editing, by extracting the ‘T cells’ and modifying the genes present in it via CRISPR technology. Haniyah is an aspiring writer and science lover. The possibilities of gene therapy hold much promise. Gene Therapy was initially meant to introduce genes straight into human cells, focusing on diseases caused by single-gene defects, such as cystic fibrosis, hemophilia, muscular dystrophy (see video 2) and sickle cell anemia (see also Wiley database on indications addressed by gene therapy clinical trials).Three types of diseases for gene therapy can be distinguished: