Gene therapy shows promise against age-related macular degeneration

According to Tufts news, a gene therapy approach using a protein called CD59, or protectin, shows promise in slowing the signs of age-related macular degeneration (AMD). A new in vivo study conducted by researchers at Tufts University School of Medicine led by Rajendra Kumar-Singh, PhD, has demonstrated for the first time that CD59 delivered by a gene therapy approach significantly reduced the uncontrolled blood vessel growth and cell death typical of AMD,which is the most common cause of blindness in the elderly.

Activation of the complement system, a part of the immune system, is responsible for slowly killing cells in the back of the eye, leading to AMD. Activation of this system leads to the generation of pores or holes known as 'membrane attack complex' or MAC in cell membranes. CD59 is known to block the formation of MAC.

Previous studies using CD59 have have had limited success, as CD59 is considered to be unstable. By continuously producing CD59 in the eye. the approach by these investigators is able to overcome these barriers, which increases hope that it can be used to fight the progression of AMD as well as other diseases.

Kumar-Singh is associate professor in the department of ophthalmology at Tufts University School of Medicine (TUSM) and member of the genetics; neuroscience; and cell, molecular, and developmental biology program faculties at the Sackler School of Graduate Biomedical Sciences at Tufts.

Kumar-Singh and colleagues delivered CD59 to the eye using a deactivated virus similar to one previously shown to be safe in humans. Using an established mouse model of age-related macular degeneration, they found that eyes treated with CD59 had 62 percent less uncontrolled blood vessel growth and 52 percent less MAC than controls.

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