Gene therapy is already known to help in treating several diseases. A new study has now revealed that they help in the treatment of life threatening cardiac disease such as Danon disease
A rare disease, Danon is a condition where the fundamental biological process of removing and recycling proteins does not work. This affects the functioning of the heart, eyes, neurologic system, liver and skeletal muscles. Patients with this type of disease require heart transplants in their third decade of life.
Science Translational Medicine published the study by researchers at UC San Diego Health. Lead researcher Eric Adler said that mutations in the gene LAMP2 caused Danon disease. The researchers have been working for nearly a decade on the disease and gene therapy.
The experiments were done in mice that missed the LAMP gene. The researchers applied Gene therapy to these mice and compared to those that did not receive treatment. Adler said that the mice that received gene showed positive results
Danon disease is common in males. In several cases, the condition is inherited by a parent typically the mother. Adler claimed that this was the first trial using gene therapy to treat cardiac disorders