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Vision for the Future: Gene Therapy is Here

SriniVas Sadda, MD

President & Chief Scientific Officer

At Doheny we are poised to begin providing patients with access to clinical trials of pioneering gene therapy any day now. It’s an advance that seems a perfect way to end the year, or begin the new one, with exciting innovation and hope for improved vision for many.

We know that many eye diseases are caused by mutations or defects in a single gene or a combination of genes. For decades, doctors have been working to find the underlying causes of these mutations– and, seeking opportunities for new treatments for a variety of diseases– ranging from retinal degenerations to optic nerve disorders.
Thanks to recent breakthrough clinical trials, we now know that the prospect of using a virus as a vector to inject new genes into the eye, works.
For example, Spark Therapeutics recently completed a Phase III clinical trial providing evidence that a gene therapy approach could restore useful vision to patients who had been blind for many years due to Leber’s Congenital Amaurosis (LCA).

After the gene was injected into one eye of patients with LCA , the demonstrated post-op recovery of vision was nothing less than marvelous: patients were able to navigate a maze using their own restored vision!
It’s the kind of improvement in vision we dream of, and we are elated at the prospect of being able to deliver treatment to our patients with previously untreatable diseases. This gene therapy treatment for LCA is now pending FDA approval

The success with the LCA approach has emboldened us to consider gene therapy for other blinding diseases. One such condition is Leber’s Hereditary Optic Neuropathy (LHON), which can lead to relatively rapid and severe loss of vision in both eyes.

We are fortunate that one of the world’s leading experts studying this condition, Alfredo Sadun, is right here at Doheny. Dr. Sadun’s laboratory has studied and uncovered many of the processes involved in the development of this condition, revealing that mutations in genes for mitochondria, the energy factories in our cells, leads to LHON.

Now Doheny will become the only eye institute on the West Coast to offer this cutting edge treatment. As Dr. Sadun is one of the pioneers in the development of new treatments for LHON, he will direct Doheny’s clinical trials for gene therapy to treat the disease.

I can’t think of a better way to wind down one year and launch into the new than with this tremendous innovation, and I thank my colleagues here at Doheny and around the world, our dedicated supporters, and patients as partners for moving us into the future.

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