Next-generation sequencing (NGS) technology has expanded in the last decades with
significant improvements in the reliability, sequencing chemistry, pipeline analyses, data …

Epidermolysis bullosa (EB) is a group of rare genetic disorders for which significant progress
has been achieved in the development of molecular therapies in the last few decades. Such …

Epidermolysis bullosa (EB) is the umbrella term for a group of rare inherited skin fragility
disorders caused by mutations in at least 20 different genes. There is no cure for any of the …

Designer nucleases allow specific and precise genomic modifications and represent
versatile molecular tools for the correction of disease-associated mutations. In this study, we …

With the ability to induce rapid and efficient repair of disease-causing mutations,
CRISPR/Cas9 technology is ideally suited for gene therapy approaches for recessively and …

Current gene-editing approaches for treatment of recessive dystrophic epidermolysis
bullosa (RDEB), an inherited, severe form of blistering skin disease, suffer from low …

Junctional epidermolysis bullosa (JEB) is a debilitating hereditary skin disorder caused by
mutations in genes encoding laminin-332, type XVII collagen (C17), and integrin-α6β4 …

End-joining‒based gene editing is frequently used for efficient reframing and knockout of
target genes. However, the associated random, unpredictable, and often heterogeneous …

Epidermolysis bullosa (EB) encompasses a heterogeneous group of inherited skin fragility
disorders, with mutations in genes encoding the basement membrane zone (BMZ) proteins …

Epidermolysis bullosa (EB) is a devastating genetic skin disease typified by a plethora of
different phenotypes and ranking from severe, early lethal, to mild localized forms. Although …