Inherited Blindness Gene Therapy Passes Next Stage


Having passed the first stage with successfully improving the eyesight in one eye of 12 young adults and children; the gene therapy for inherited blindness has now passed its second tests on animals to decipher whether or not it is safe to conduct the therapy in the other untreated eye. Therefore, patients could soon be receiving gene therapy in not one but both of their eyes.

The tests have been conducted by the University of Pennsylvania School of Medicine alongside The Children’s Hospital of Philadelphia and the results can be viewed in the latest edition of Science Translational Medicine. The study entailed an examination in four monkeys and six dogs who had received the gene therapy in both eyes. The animals had one injection of the gene therapy in their right eyes before being administered the same injection in the left eye two weeks later. The injection contains genes that are used to restore retinal light receptors with the use of a vector to deliver the genes. Scientists monitored the animals for any adverse side effects; e.g. toxic effects. Not only did the animals have no side effects whatsoever but they also had dramatically improved eyesight. Having been bred specifically to have congenital blindness, the dogs improved eyesight shows a dramatic pioneering landmark in gene therapy.

The current gene therapy under investigation has been designed to treat Leber’s Congenital Amaurosis (LCA) which is an inherited disease of the retina that can lead to the sufferer going completely blind before they reach adulthood. The concerns had stemmed from the gene therapy’s use of a virus (the vector that delivers the gene) as previous gene therapy for hemophilia (a bleeding disorder) used this particular virus, adeno-associated virus (AAV) which consequently caused an immune response which prevented the gene therapy’s benefits from continuing. Scientists are positive that in this particular study, the body’s natural antibodies that try to fight AAV will not affect the gene therapy’s benefits.

7th March 2010

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