Gene Therapy, new hope for Patients with Hereditary Lung Disease

Science daily reported on august 11th 2009 stated that the Researchers at the University of Massachusetts Medical School and the University of Florida in Gainesville has done wonderful job in the gene therapy trial of boosting protective protein in patients with hereditary lung disease. They have proved to be successful by giving new functional genes to patients who have this hereditary gene problem and the results are positive. This is very good news for people suffering from hereditary lung disease and also this news will bring a good hope in those people. The most interesting news about this therapy is that with just one series of injections our bodies will be able to produce the alpha-1 protein we need till our life time unlike the current existing therapies. The main reason for hereditary lung disease is due to the deficiency of alpha-1 antitrypsin. The people suffering from deficiency of alpha-1 antitrypsin are easy to get affected by any allergies from air as they are not able to produce protein alpha-1 antitrypsin which is normally produced by the liver to protect the various infections that affect the lungs.

The clinical test was practiced in few patients who were affected by this deficiency. An injection of alpha-1protein was given to them in their deltoid muscle in their upper arm. Till one year they were able to produce noticeable amount of alpha-1 antitrypsin. Mark L. Brantley, MD, at UF's College of Medicine he is the professor of medicine, molecular genetics and microbiology in addition to that he is the first author of the study who said the deltoid muscle where the injection is given acts as a factory for making the protein that these individuals are missing. Later as a part of their research they divided nine patients into three groups and were given the adeno associated virus which helps in production of alpha-1 protein. All nine Patients were given injections in their upper arms deltoid muscle. Nine injections were given to them .in each group dosage given was varying. These tests took place at the General Clinical Research Center at Shands at UF Medical Center. After one year the replaced gene was doing its work in three patients successfully who were given the maximum dosage.

The researchers were very happy as there was no rejection of the transferred gene in the patient’s body or the protein that is created newly, though the patients showed some high immune response therapy. That is very good development in gene therapy said Brantley. In the UF Genetics Institute he is a member of the Powell Gene Therapy Center and also he is the Alpha-1 Foundation Research Professor at UF and is a consultant for the organization. Walsh said “alpha-1 community is incredibly grateful for the progress that these dedicated investigators have made” Walsh. The Alpha-1 Foundation’s president and chief executive officer is Mr. Walsh and also he has been constantly supporting various researchers and investigations in gene therapy field for many years. Though currently there is some effective injection of alpha-1 protein derived from human plasma for serious breathing symptoms. They don’t really cure the disease they only slow its progression. So the American Lung Association says that the patients should continue the injections throughout their life. So definitely this research is a boon o many people suffering from serious lungs related problems.