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Experimental Cystic Fibrosis Treatment Uses Improved Gene Therapy Techniques

Posted on November 17, 2015 by Disability Help Group

Gene therapy has changed the course of treatment for several disabling conditions such as congenital blindness and hemophilia. However, the technology is still in early stages of use in medical care, and doctors have yet to find a successful method of implementation for cystic fibrosis. This chronic disease causes complications of the respiratory and digestive systems, which can lead to breathing difficulties and poor weight gain, especially in children.

Currently, cystic fibrosis symptoms can only be managed, not cured, and previous attempts at gene therapy treatments have been only marginally positive. Now, new research into an altered method of gene therapy is showing promise in treating animal and laboratory models of the disease.

Researchers KU Leuven in Belgium focused their work on the CFTR, the faulty gene responsible for causing cystic fibrosis. Using the new gene therapy known as “recombinant adeno-associated virus (rAAV)”, the researchers were able to swap in a healthy CFTR gene through the use of a relatively harmless AAV virus as the vehicle. Mice with cystic fibrosis genes that were given the virus, introduced through their airways, showed an improvement in breathing ability.

The rAAV therapy was also used on human gut cell cultures in laboratory tests. The cultures showed improved chloride and fluid transport, making researchers hopeful that eventual human clinical trials could also be successful. However, the research team is cautious to suggest that this could be a potential path to a cure, as much more research is necessary before human clinical trials can even be developed.

Cystic Fibrosis Can Cause Disability from Birth

If your child was born with cystic fibrosis, Disability Help Group can assist you in applying for Social Security Disability Insurance (SSDI) and Supplemental Security Income (SSI) benefits. Contact us online or call us at 1-(800)-800-3332 to schedule a consultation with our disability advocates!