Publications by authors named "Thuylinh Michelle Nguyen"
Article Synopsis
- DEBCT is a new cell therapy that creates skin grafts from patients’ own iPS cells to treat Dystrophic Epidermolysis Bullosa (DEB).
- This method combines CRISPR gene editing with cell reprogramming for faster production of corrected cells, leading to effective and diverse skin cell types.
- Early studies show that these grafts are safe and can effectively improve skin conditions in DEB patients within a month of treatment.
Background: 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.
Methods: We developed Dystrophic Epidermolysis Bullosa Cell Therapy (DEBCT), a new generation GMP-compatible (cGMP), reproducible, and scalable platform to produce autologous clinical-grade iPS cell-derived organotypic induced skin composite (iSC) grafts to treat incurable wounds of patients lacking type VII collagen (C7).