The current standard treatment for some forms of AMD requires an injection directly into the eye approximately every four weeks. This finding is especially encouraging because it would allow for a safer and more convenient route of administration of treatment,” says co-first author Siobhan Cashman, PhD, an assistant professor in the ophthalmology department. “Treatment was effective when administered at a very specific location beneath the retina, but importantly, also when it was administered to the center of the eye.
#HEMERA BIOSCIENCES MAC#
Using an established mouse model of age-related macular degeneration, they found that eyes treated with CD59 had 62% less uncontrolled blood vessel growth and 52% less MAC than controls. Kumar-Singh and colleagues delivered CD59 to the eye using a deactivated virus similar to one previously shown to be safe in humans. Kumar-Singh is an associate professor in the ophthalmology department at Tufts University School of Medicine and member of the genetics neuroscience and cell, molecular, and developmental biology program faculties at the Sackler School of Graduate Biomedical Sciences at Tufts. The gene therapy approach that we developed continuously produces CD59 in the eye and overcomes these barriers, giving us renewed hope that it can be used to fight the progression of AMD and potentially other diseases,” senior author Rajendra Kumar-Singh, PhD, says. “CD59 is unstable and hence previous studies using CD59 have had limited success.
CD59 is known to block the formation of MAC. Activation of this system leads to the generation of pores or holes known as ‘membrane attack complex’ or MAC in cell membranes. The study was published in PLoS ONE.Īctivation 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. A gene therapy approach using a protein called CD59, or protectin, shows promise in slowing the signs of age-related macular degeneration (AMD), according to a new in vivo study by researchers at Tufts University School of Medicine.The researchers 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.
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