CRISPR–Cas9 is the state-of-the-art programmable genome-editing tool widely used in
many areas. For safe therapeutic applications in clinical medicine, its off-target effect must …

Cancer is a devastating condition characterised by the uncontrolled division of cells with
many forms remaining resistant to current treatment. A hallmark of cancer is the gradual …

Undesired on-and off-target effects of CRISPR-Cas nucleases remain a challenge in
genome editing. While the use of Cas9 nickases has been shown to minimize off-target …

Homology-directed recombination (HDR) between donor constructs and acceptor genomic
sequences cleaved by programmable nucleases, permits installing large genomic edits in …

Recessive dystrophic epidermolysis bullosa, a devastating skin fragility disease
characterized by recurrent skin blistering, scarring, and a high risk of developing squamous …

The monogenetic disease epidermolysis bullosa (EB) is characterised by the formation of
extended blisters and lesions on the patient's skin upon minimal mechanical stress. Causal …

Genodermatoses encompass a wide range of inherited skin diseases, many of which are
monogenic. Genodermatoses range in severity and result in early-onset cancers or life …

Genome-editing technologies that enable the introduction of precise changes in DNA
sequences have the potential to lead to a new class of treatments for genetic diseases …

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 …