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      Vectors and Gene Delivery to the Retina

      1 , 2 , 3 , 1 , 2 , 3
      Annual Review of Vision Science
      Annual Reviews

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          AAV-Mediated Anterograde Transsynaptic Tagging: Mapping Corticocollicular Input-Defined Neural Pathways for Defense Behaviors.

          To decipher neural circuits underlying brain functions, viral tracers are widely applied to map input and output connectivity of neuronal populations. Despite the successful application of retrograde transsynaptic viruses for identifying presynaptic neurons of transduced neurons, analogous anterograde transsynaptic tools for tagging postsynaptically targeted neurons remain under development. Here, we discovered that adeno-associated viruses (AAV1 and AAV9) exhibit anterograde transsynaptic spread properties. AAV1-Cre from transduced presynaptic neurons effectively and specifically drives Cre-dependent transgene expression in selected postsynaptic neuronal targets, thus allowing axonal tracing and functional manipulations of the latter input-defined neuronal population. Its application in superior colliculus (SC) reveals that SC neuron subpopulations receiving corticocollicular projections from auditory and visual cortex specifically drive flight and freezing, two different types of defense behavior, respectively. Together with an intersectional approach, AAV-mediated anterograde transsynaptic tagging can categorize neurons by their inputs and molecular identity, and allow forward screening of distinct functional neural pathways embedded in complex brain circuits.
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            Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics.

            Considered by some to be among the simpler forms of life, viruses represent highly evolved natural vectors for the transfer of foreign genetic information into cells. This attribute has led to extensive attempts to engineer recombinant viral vectors for the delivery of therapeutic genes into diseased tissues. While substantial progress has been made, and some clinical successes are over the horizon, further vector refinement and/or development is required before gene therapy will become standard care for any individual disorder.
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              Genetic reactivation of cone photoreceptors restores visual responses in retinitis pigmentosa.

              Retinitis pigmentosa refers to a diverse group of hereditary diseases that lead to incurable blindness, affecting two million people worldwide. As a common pathology, rod photoreceptors die early, whereas light-insensitive, morphologically altered cone photoreceptors persist longer. It is unknown if these cones are accessible for therapeutic intervention. Here, we show that expression of archaebacterial halorhodopsin in light-insensitive cones can substitute for the native phototransduction cascade and restore light sensitivity in mouse models of retinitis pigmentosa. Resensitized photoreceptors activate all retinal cone pathways, drive sophisticated retinal circuit functions (including directional selectivity), activate cortical circuits, and mediate visually guided behaviors. Using human ex vivo retinas, we show that halorhodopsin can reactivate light-insensitive human photoreceptors. Finally, we identified blind patients with persisting, light-insensitive cones for potential halorhodopsin-based therapy.
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                Author and article information

                Journal
                Annual Review of Vision Science
                Annu. Rev. Vis. Sci.
                Annual Reviews
                2374-4642
                2374-4650
                September 15 2017
                September 15 2017
                : 3
                : 1
                : 121-140
                Affiliations
                [1 ]Inserm, Institut de la Vision, UMR S968, 75012 Paris, France;,
                [2 ]Sorbonne Universités, UPMC Université Paris 6, UMR S968, 75012 Paris, France
                [3 ]CNRS, UMR 7210, 75012 Paris, France
                Article
                10.1146/annurev-vision-102016-061413
                28937950
                dc7a840e-769b-44b1-8faa-cbbd48adae1f
                © 2017
                History

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