Article
Intravitreal delivery of a novel AAV vector targets ON bipolar cells and restores visual function in a mouse model of complete congenital stationary night blindness.
Human molecular genetics - 1 Nov 2015
Scalabrino Miranda L, Boye Sanford L, Fransen Kathryn M H, Noel Jennifer M, Dyka Frank M, Min Seok Hong, Ruan Qing, De Leeuw Charles N, Simpson Elizabeth M, Gregg Ronald G, McCall Maureen A, Peachey Neal S, Boye Shannon E
Abstract excerpt
Adeno-associated virus (AAV) effectively targets therapeutic genes to photoreceptors, pigment epithelia, Müller glia and ganglion cells of the retina. To date, no one has shown the ability to correct, with gene replacement, an inherent defect in bipolar cells (BCs), the excitatory interneurons of the retina. Targeting BCs with gene replacement has been difficult primarily due to the relative inaccessibility of...
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