Lipid nanoparticles efficiently deliver the base editor ABE8e for COL7A1 correction in dystrophic epidermolysis bullosa fibroblasts in vitro.

Lipid nanoparticles (LNPs) have been widely approved and used on a global scale for delivery of mRNA. LNPs can package and deliver mRNA encoding gene editors, including adenine base editors (ABEs), which convert A •T base pairs to G•C base pairs without double-stranded DNA breaks or donor DNA (Gaudelli et al. 2017). ABE is a potential treatment approach for the inherited blistering disease dystrophic epidermolysis bullosa (DEB). DEB results from pathogenic variants in COL7A1, leading to dysfunctional or a bsent type VII collagen (C7), a major component of anchoring fibrils (AFs) that adhere the dermal-epidermal junction (DEJ), giving stability to skin (Bardhan et al.
Source: Journal of Investigative Dermatology - Category: Dermatology Authors: Tags: Letters to the Editor Source Type: research