Post-injury delivery of rAAV2-CNTF combined with short-term pharmacotherapy is neuroprotective and promotes extensive axonal regeneration after optic nerve trauma.
作者： Mats Hellström , Margaret A. Pollett , Alan R. Harvey
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摘要: Abstract Recombinant adeno-associated viral (rAAV) vectors expressing neurotrophic genes reduce neuronal death and promote axonal regeneration in central nervous system (CNS) injury models. Currently, however, use of rAAV to treat clinical neurotrauma is problematic because there a delay the onset transgene expression. Using adult rat retina optic nerve (ON), we have tested whether gene therapy administered at time combined with short-term pharmacotherapy has synergistic effects that enhance survival regeneration. The ON was transected 1.5 cm segment autologous peripheral (PN) grafted onto cut end. At this time, bicistronic rAAV2 encoding ciliary factor (CNTF) green fluorescent protein (rAAV2-CNTF-GFP) injected into injured eye. To provide interim support for axotomized retinal ganglion cells (RGCs) during vector integration therapeutic expression, rCNTF cyclic adenosine monophospha...
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