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Local gene therapy in vivo for recessive dystrophic epidermolysis bullosa

 Last Update: 2022-04-24
 Source: Internet
 Author: User

Summary

Recessive dystrophic epidermolysis bullosa (RDEB) is a lifelong inherited skin disorder associated with blisters, lesions, and scarring caused by mutations in the COL7A1 gene, which encodes the anchoring fibrous component, collagen VII (C7)

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Recessive dystrophic epidermolysis bullosa (RDEB) is a devastating rare inherited skin disease with an incidence of 135 per million in the United States

Many early C7 replacement strategies have been evaluated for RDEB8

In vivo direct gene transfer for the treatment of inherited skin diseases has been a long-standing unrealized goal in the field of gene therapy

The large ~9 kb COL7A1 transgene required for RDEB skin correction presents additional challenges for vector engineering

We describe here the development and clinical translation of a localized gene therapy for RDEB that can be reapplied without serious adverse events

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