Gene Therapy Focusing on Hemophilia

Gene therapy is focused on haemophilia gene therapy to reinsert specific genes useful for the treatment of hereditary diseases. There are three basic forms of this gene therapy. The first is gene inactivation therapy, the introduced gene neutralizes the protein and balances the amount or elimination of defective proteins. Another type is gene enhancement therapy which inserts the original form of the gene or the normal form of the gene into the chromosome of the cell. This procedure is commonly used when genes containing small amounts of active or deletion genes are responsible for genetic disorders.

Scientists are conducting research called genome therapy for hemophilia patients. Gene therapy is an experimental technique that attempts to provide genetic information that the body does not have to the body. Because hemophilia is caused only by defective genes, it is considered a good test for gene therapy. Scientists hope that hemophilia patients will be able to provide the genetic information they need to create coagulation factors that they miss.

Turning to the future of gene therapy. . The first companies targeting specific genetic diseases need to determine the optimal MED for viral treatment. In the case of haemophilia, gene therapy must increase the level of Factor VII protein in the blood to a significant extent to the bleeding event, and it may be eliminated. In addition, this result must occur without toxic reactions, ie liver enzyme peaks (ALT).

BioMarin is a company specialized in gene therapy that replaces the most common types of haemophilia defective genes and effectively cures disease. Last December, the company announced early clinical trial results showing a significant increase in blood clotting protein in hemophilia in nine patients treated. After a year and a half treatment, the patient has less bleeding problems and can reduce the injection of coagulation factors. At the same time, few patients with haemophilia B, a rare disorder, have received amazing treatment after a single treatment.

New gene therapy developed by Philadelphia Children's Hospital has brought very promising results. Preliminary studies have shown that monotherapy with experimental treatment may be able to help patients with hemophilia B lack coagulation factor IX. Treatment involves the use of genes designed to replace incorrect genes in hemophilia patients. Put genetically engineered genes in inactivated viruses and inject them into the liver to help patients produce clotting factors and prevent bleeding. Dr. Lindsay George of Philadelphia Children's Hospital says: