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Gene therapy can help blindness and other conditions

Posted on 05-13-16 in Behavioral Health, Research, Treatment

Gene therapy can help blindness and other conditions

Gene therapy, an experimental medical technique still in its very early stages, may offer hope to some people with previously incurable conditions – particularly when combined with other treatment modalities.

Case in point: A blind woman in Texas with retinitis pigmentosa – an inherited, degenerative eye disease that causes blindness – is participating in a revolutionary clinical trial involving a combination of gene therapy and light that could potentially restore her eyesight. Although this case isn’t connected to psychology or addiction treatment, the combination of treatment may have benefits for patients in those areas.

How does the treatment work?

Retinitis pigmentosa blinds patients by causing the retina’s photoreceptor cells to die off. Optogenetics – the therapy the blind woman and others in the clinical trial are receiving – attempts to restore eyesight by introducing a new photosensitive gene to retinal cells to restore their light-sensing ability.

According to the study’s sponsor, Michigan-based RetroSense Therapeutics, a photosensitivity gene derived from algae called channelrhodopsin-2 is injected into retinal cells called ganglions. The new gene should start making more of the light-sensing protein from the injection, causing them to respond to light.

CEO Sean Ainsworth cautioned the MIT Technology Review that successful results would be far from full sight for the patients – he hopes the treatment will allow patients to read large type or “see tables and chairs.” However, a successful result might mean much for research in other fields. “This is going to be a gold mine of information about doing optogenetics studies in humans,” said National Institute on Drug Abuse scientific director Antonello Bonci, M.D., in a Technology Review article.

Potential treatment roles for gene therapy

A World Journal of Biological Psychiatry paper published in 2011 outlined several ways gene therapy could be used to treat addiction, psychosis and other mental disorders. For example, in 2008, studies done on rats showed less of a desire to drink after receiving an injection of alcohol-inhibiting genes. Other studies on rodents have shown potential for gene therapy as a potentially effective treatment method.

In 2013, a study published in the Lancet traced the same gene variations to five major mental illnesses: attention deficit hyperactivity disorder (ADHD), autism, bipolar disorder, major depressive disorder and schizophrenia. Researchers examined more than 33,000 patients and found the regulation of calcium flow in brain cells might play a role in how the disorders develop, suggesting a role for gene therapy in treating those disorders.

What is gene therapy?

According to the American Cancer Society, genes are pieces of deoxyribonucleic acid (DNA) found inside every cell. Each gene contains a DNA sequence, which contains a code to make a protein. Each protein has a particular function or job in the body.

Gene therapy is an experimental medical technique that uses genes to treat or prevent diseases. The U.S. National Library of Medicine’s Genetics Home Reference site reports that gene therapy could play different roles in medicine, including:

  • Deactivating mutated genes that aren’t functioning correctly
  • Giving the body new genes to help fight diseases
  • Replacing mutated genes with a healthy copy of the gene

The library reports that while gene therapy shows promise in treating certain diseases, the technique is still being studied and currently only being tested with diseases that have no other cures.

Gene therapy is still very far off as a widespread, proven tool to address mental illness and addiction. However, it’s not necessary to wait – many mental disorders respond to treatment methods like cognitive behavioral therapy and other psychotherapies. Sovereign Health is a leading treatment provider dedicated to using proven, scientifically backed methods to ensure a lasting recovery for its patients. Contact our 24/7 helpline for more details.

About the author

Brian Moore is a staff writer and graphic designer for the Sovereign Health Group. A 20-year veteran of the newspaper industry, he writes articles and creates graphics across Sovereign’s portfolio of marketing and content products. Brian enjoys music, bicycling and playing the tuba, which’s he’s done with varying degrees of success for over 25 years. For more information and other inquiries about this media, contact the author and designer at