Promising results of gene therapy to treat diseases of the eye

The easy accessibility of the eye and the established link between specific genetic defects and ocular disorders offer hope for using gene therapy to provide long-term therapeutic benefit. Two reports in the current issue of Human Gene Therapy, a peer-reviewed journal published by Mary Ann Liebert, Inc., describe the effective replacement of a human gene to preserve photoreceptor function in a mouse model of severe retinal degeneration.

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