作者
Shaundra Eichstadt, Melissa Barriga, Anusha Ponakala, Claudia Teng, Ngon T Nguyen, Zurab Siprashvili, Jaron Nazaroff, Emily S Gorell, Albert S Chiou, Lisa Taylor, Phuong Khuu, Douglas R Keene, Kerri Rieger, Rohit K Khosla, Louise K Furukawa, H Peter Lorenz, M Peter Marinkovich, Jean Y Tang
发表日期
2019/10/10
期刊
JCI insight
卷号
4
期号
19
出版商
American Society for Clinical Investigation
简介
BACKGROUND
Recessive dystrophic epidermolysis bullosa (RDEB) patients have mutations in the COL7A1 gene and thus lack functional type VII collagen (C7) protein; they have marked skin fragility and blistering. This single-center phase 1/2a open-label study evaluated the long-term efficacy, safety, and patient-reported outcomes in RDEB patients treated with gene-corrected autologous cell therapy.
METHODS
Autologous keratinocytes were isolated from participant skin biopsies. Epidermal sheets were prepared from cells transduced with a retrovirus carrying the full-length human COL7A1 gene. These gene-corrected autologous epidermal sheets measured 5× 7 cm (35 cm 2) and were transplanted onto 6 wound sites in each of 7 adult participants (n= 42 sites total) from 2013 to 2017. Participants were followed for 2 to 5 years.
RESULTS
No participants experienced any serious related adverse events. Wound …
学术搜索中的文章
S Eichstadt, M Barriga, A Ponakala, C Teng… - JCI insight, 2019