可扩展和 cGMP 兼容的自体器官型细胞疗法治疗营养不良性大疱性表皮松解症。
Mesh : Humans Epidermolysis Bullosa Dystrophica / therapy genetics Animals Induced Pluripotent Stem Cells / transplantation metabolism cytology Mice Collagen Type VII / genetics metabolism Cell- and Tissue-Based Therapy / methods Fibroblasts / metabolism Cell Differentiation Keratinocytes / metabolism transplantation Skin / metabolism Transplantation, Autologous Male Mutation Female Skin Transplantation / methods Gene Editing / methods CRISPR-Cas Systems
来 源:
DOI:10.1038/s41467-024-49400-z
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