In hemophilia, one of the clotting factor proteins normally needed for the blood to clot does not work properly, either because the protein itself is abnormal, or because too little or none of it is being produced.In the vast majority of cases, hemophilia is caused by mutations in a gene ...
The method of treatment is another con when it comes to gene therapy for hemophilia. In order to get the genetic material to the cells, it is necessary to attach it to viruses, which are then injected into the body so they can invade those cells. In some cases, this can trigger an im...
Treatment depends on the type and severity of hemophilia. The treatment aims to prevent bleeding episodes mainly inside the head and the joints. The use of gene therapy to replace the defective genes is currently under study. Blood transfusions may be required in case of significant blood loss....
A) What is the cause of Duchenne Muscular Dystrophy (DMD)? B) Why does this disease only affect males? C) What happens each time the muscle contracts in a child with DMD? Which of the diseases is caused by a faulty chromosome number? A. goiter B. Syphilis C. Influenza D. Mongoloid ...
the company has also been testing a way to combat hemophilia B, a blood clotting disorder, by intravenous treatment with viruses carrying a corrected version of the gene that codes for a blood-clotting protein called factor IX. This is one to watch because the disease, which affects one in ...
These therapies are intended to treat monogenic diseases, which are disease states caused by 1 missing or faulty gene or gene pair. Monogenic diseases include conditions such as cystic fibrosis, Duchenne muscular dystrophy, amyotrophic lateral sclerosis (ALS), hemophilia, Rett syndrome, and SMA. ...
the company has also been testing a way to combat hemophilia B, a blood clotting disorder, by intravenous treatment with viruses carrying a corrected version of the gene that codes for a blood-clotting protein called factor IX. This is one to watch because the disease, which affects one in ...