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

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...

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Autors principals:	Alex du Rand, John Hunt, Christopher Samson, Evert Loef, Chloe Malhi, Sarah Meidinger, Chun‐Jen Jennifer Chen, Ashley Nutsford, John Taylor, Rod Dunbar, Diana Purvis, Vaughan Feisst, Hilary Sheppard
Format:	Artigo
Idioma:	Inglês
Publicat:	Wiley 2024-07-01
Col·lecció:	Bioengineering & Translational Medicine
Matèries:	CRISPR/Cas9 exon skipping gene therapy recessive dystrophic epidermolysis bullosa (RDEB) ribonucleoproteins structural variants
Accés en línia:	https://doi.org/10.1002/btm2.10640
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