Highly efficient biallelic correction of homozygous COL7A1 mutation using ABE8e adenine base editor.

Researchers have made progress in developing a gene repair approach for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), a blistering skin disease. They used a technique called base editing, which avoids the risk of DNA breaks associated with other gene editing methods. The researchers achieved high efficiency in correcting a specific mutation in the COL7A1 gene, which is associated with RDEB, using a novel form of adenine base editor. They also explored the use of lipid delivery systems for delivering the gene editing components. This research shows promise for developing new therapies for RDEB and other skin diseases. [Extracted from the article]