Features of the Week
Glowing proof of gene therapy delivered to the eye, by Dr. Francis Collins, NIH Director, Jul 11
Gene therapy trial 'cures children', by James Gallagher Health and science reporter, BBC News, Jul 11
New stem cell gene therapy success, by Dr Philippa Brice, PHG Foundation, Jul 11
Gene therapy coming of age? Using lentiviral vectors to replace mutated genes in blood stem cells, scientists successfully treat two rare diseases apparently without causing harmful side effects, by Dan Cossins, The Scientist, Jul 11
Health researchers hack HIV to cure children with rare genetic disorders, Circa blog, Jul 13
Scientists based at Berkeley have engineered a virus that can carry healthy genes through the jelly-like substance in the eye to reach the cells that make up the retina—the back of the eye that detects light.
Current gene therapy techniques deliver therapeutic viruses using a needle that must pierce the retina to deliver its cargo. It’s a risky procedure because it can cause retinal detachment, potentially making the condition worse. The NIH-funded researchers sifted through hundreds of millions of newly engineered viruses to identify one they called a 7M8, which had the right properties to penetrate the cells of the retina. Their ultimate goal is to cure two types of hereditary blindness—X-linked retinoschisis (which only affects boys) and Leber’s congenital amaurosis.
When the researchers tested 7M8 in a healthy primate, the virus delivered genes to the retina and to the fovea—a hard-to-penetrate region of the retina responsible for fine-scale vision. You can see this in the picture: the green is glowing proof that the virus successfully delivered its genetic cargo into the photoreceptor cells of the retina.
It’s early days, but this is a significant step in the development of new therapies that will restore sight to people with both inherited and age-related forms of blindness.
In vivo-directed evolution of a new adeno-associated virus for therapeutic outer retinal gene delivery from the vitreous. Dalkara D, Byrne LC, Klimczak RR, Visel M, Yin L, Merigan WH, Flannery JG, Schaffer DV. Sci Transl Med. 2013 Jun 12
NIH support: Nanomedicine Development Center for the Optical Control of Biological Function (Common Fund)