Article

A scalable, GMP-compatible, autologous organotypic cell therapy for Dystrophic Epidermolysis Bullosa

2023-03-01

Abstract excerpt

<h4>Summary</h4> <h4>Background</h4> Gene editing in induced pluripotent stem (iPS) cells has been hailed to enable new cell therapies for various monogenetic diseases including dystrophic epidermolysis bullosa (DEB). However, manufacturing, efficacy and safety roadblocks have limited the development of genetically corrected, autologous iPS cell-based therapies. <h4>Methods</h4> We developed Dystrophic Epidermo...

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Literature Corpus work: 198fb828-5425-5154-a170-a8cbbf017299
DOI: 10.1101/2023.02.28.529447

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