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Gene Therapy: Location, Location, Location

This article was originally published in Start Up

Executive Summary

The new gene therapy start-ups are learning to live with the technology’s limitations--and even to exploit them. While systemic treatments have generally failed, largely because doctors couldn’t deliver high-enough and frequent-enough doses to overcome the relatively low transfection rates and short-term expression, new companies are targeting localized diseases, in vascular medicine, wound care and certain neurological conditions, in which treatment doesn’t require long-term expression and where direct delivery of genes maximizes the effective dose.

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