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- W4311448265 abstract "Dystrophic epidermolysis bullosa is a genetic disorder in which there is a loss of anchoring fibrils and structural integrity in the superficial layers of the dermis owing to mutations in the gene encoding type VII collagen (COL7A1), as explained by Payne in this issue of the Journal.1 This process leads to the development of painful skin blisters along with a range of secondary illnesses.2 The condition can be inherited in dominant or recessive patterns and in varying degrees of severity. There are currently no approved therapies for the disorder. Gene therapy has made strides in recent years, and in a . . ." @default.
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- W4311448265 date "2022-12-15" @default.
- W4311448265 modified "2023-10-17" @default.
- W4311448265 title "The Coming of Age of Topical Gene Therapy for Dystrophic Epidermolysis Bullosa" @default.
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- W4311448265 doi "https://doi.org/10.1056/nejme2212899" @default.
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