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

Gernot NeumayerJessica L TorkelsonShengdi LiKelly McCarthyHanson H ZhenMadhuri VangipuramJoanna JackowAvina RamiCorey HansenZongyou GuoSadhana GaddamAlberto PappalardoLingjie LiAmber CramerKevin R RoyThuylinh Michelle NguyenKoji TanabePatrick S McGrathAnna BrucknerGanna BilousovaDennis RoopIrene BaileyJean Y TangAngela ChristianoLars M SteinmetzMarius WernigAnthony E Oro

Published in: bioRxiv : the preprint server for biology (2023)

DEBCT successfully overcomes previous roadblocks and represents a robust, scalable, and safe cGMP manufacturing platform for production of a CRISPR-corrected autologous organotypic skin graft to heal DEB patient wounds.

Keyphrases

cell therapy
bone marrow
platelet rich plasma
crispr cas
nitric oxide
single cell
wound healing
case report
genome editing
genome wide
stem cells
mesenchymal stem cells
soft tissue
protein kinase
escherichia coli
gene expression
staphylococcus aureus