Gene Therapy May Lead to Cure for Macular Degeneration

With the discovery of a gene therapy that increases light sensitivity and improves vision in patients who were virtually blind.  Will this gene therapy could lead to a cure for blindness and a cure for macular degeneration.

Leber’s congenital amaurosis (LCA) is a rare eye disease that is inherited at birth whe nboth parents are carriers of a defective gene known to cause the disorder.  This eye disorder prevents the retina from processing light and it usually appears in people at birth or shortly after birth.

Researchers at the Children’s Hospital of Philadelphia injected a normal gene into a single eye of three patients suffering from LCA, leaving the other eye untouched.  Over the course of six-months, all patients (one 19, the other two 26) showed continual improvement.

According to Dr. Jean Bennett of the University of Pennsylvania and one of the studies authors, “All patients could read at least four lines on the eye chart.”  None of the patients injected with the gene showed any adverse effects from the treatment but showed considerable improvement in their eye disease.

In a parallel British study, only one of the patients responded favourably to the treatment.  That patient found that he could navigate through a maze more easily than he could prior to the treatment.  The other two patients showed no significant improvement in their vision.

The researchers hope that these advances in gene therapy could pave the way for finding better treatments and possibly even cures for other eye diseases such as age-related macular degeneration.

The results of both studies were published in the New England Journal of Medicine.

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