Dystrophic epidermolysis bullosa (DEB) is one of the major types of EB, a rare hereditary
group of trauma-induced blistering skin disorders. DEB is caused by inherited pathogenic …

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 …

Spliceosome‐mediated RNA trans‐splicing, or SMaRT, is a promising strategy to design
innovative gene therapy solutions for currently intractable genetic diseases. SMaRT relies …

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

The promising clinical results obtained for ocular gene therapy in recent years have paved
the way for gene supplementation to treat recessively inherited forms of retinal …

Recessive dystrophic epidermolysis bullosa (RDEB) is a rare genetic disease resulting from
inadequate type VII collagen (C7). Although recurrent skin blisters and wounds are the most …

Background: Epidermolysis bullosa (EB) is a clinically-heterogeneous genodermatosis with
severe manifestations in the skin and other organs. The significant burden this condition …

RNA trans-splicing represents an auspicious option for the correction of genetic mutations at
RNA level. Mutations within COL7A1 causing strong reduction or absence of type VII …

RNA trans-splicing has become a versatile tool in the gene therapy of monogenetic
diseases. This technique is especially valuable for the correction of mutations in large genes …

Mutations within the COL7A1 gene underlie the inherited recessive subtype of the blistering
skin disease dystrophic epidermolysis bullosa (RDEB). Although gene replacement …