Highly efficient CRISPR/Cas9‐mediated exon skipping for recessive dystrophic epidermolysis bullosa

• Main Authors: Alex du Rand (Author), John Hunt (Author), Christopher Samson (Author), Evert Loef (Author), Chloe Malhi (Author), Sarah Meidinger (Author), Chun‐Jen Jennifer Chen (Author), Ashley Nutsford (Author), John Taylor (Author), Rod Dunbar (Author), Diana Purvis (Author), Vaughan Feisst (Author), Hilary Sheppard (Author)
• Format: Book
• Published: Wiley, 2024-07-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Abstract Gene therapy based on the CRISPR/Cas9 system has emerged as a promising strategy for treating the monogenic fragile skin disorder recessive dystrophic epidermolysis bullosa (RDEB). With this approach problematic wounds could be grafted with gene edited, patient‐specific skin equivalents. Pr...