Synaptic pathology and therapeutic repair in adult retinoschisis mouse by AAV-RS1 transfer

Strategies aimed at invoking synaptic plasticity have therapeutic potential for several neurological conditions. The human retinal synaptic disease X-linked retinoschisis (XLRS) is characterized by impaired visual signal transmission through the retina and progressive visual acuity loss, and mice la...

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Veröffentlicht in:	The Journal of clinical investigation 2015-07, Vol.125 (7), p.2891-2903
Hauptverfasser:	Ou, Jingxing, Vijayasarathy, Camasamudram, Ziccardi, Lucia, Chen, Shan, Zeng, Yong, Marangoni, Dario, Pope, Jodie G, Bush, Ronald A, Wu, Zhijian, Li, Wei, Sieving, Paul A
Format:	Artikel
Sprache:	eng
Schlagworte:	Adeno-associated virus Animals Biomedical research Calcium Signaling Cell Adhesion Molecules - deficiency Cell Adhesion Molecules - genetics Cell Adhesion Molecules - metabolism Dependovirus - genetics Disease Models, Animal Dopamine Electroretinography Eye Proteins - genetics Eye Proteins - metabolism Gene therapy Genetic Therapy - methods Genetic Vectors Humans Immunoglobulins Infrared imaging systems Male Membranes Mice Mice, Inbred C57BL Mice, Knockout Neuronal Plasticity Pathology Photoreceptor Cells, Vertebrate - metabolism Photoreceptor Cells, Vertebrate - pathology Photoreceptors Proteins Receptors, Metabotropic Glutamate - metabolism Retina Retinoschisis - genetics Retinoschisis - pathology Retinoschisis - therapy Synapses - metabolism Synapses - pathology TRPM Cation Channels - metabolism
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