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AAV2 vector optimization for retinal ganglion cell-targeted delivery of therapeutic genes - Gene Therapy

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AAV2 vector optimization for retinal ganglion cell-targeted delivery of therapeutic genes - Gene Therapy

Recombinant adeno-associated virus (AAV)-2 has significant potential as a delivery vehicle of therapeutic genes to retinal ganglion cells (RGCs), which are key interventional targets in optic neuropathies. Here we show that when injected intravitreally, AAV2 engineered with a reporter gene driven by cytomegalovirus (CMV) enhancer and chicken β-actin (CBA) promoters, displays ubiquitous and high RGC expression, similar to its synthetic derivative AAV8BP2.

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AAV2 vector optimization for retinal ganglion cell-targeted delivery of therapeutic genes - Gene Therapy

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AAV2 vector optimization for retinal ganglion cell-targeted delivery of therapeutic genes - Gene Therapy

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Unlocking the Power of Knowledge
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Unlocking the Power of Knowledge
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