Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated
protein 9 (Cas9) gene-editing technology is the ideal tool of the future for treating diseases …

The growing clinical success of hematopoietic stem/progenitor cell (HSPC) gene therapy
(GT) relies on the development of viral vectors as portable" Trojan horses" for safe and …

Adenine base editors (ABEs) catalyze specific A-to-G conversions at genomic sites of
interest. However, ABEs also induce cytosine deamination at the target site. To reduce the …

Clustered regularly interspaced short palindromic repeats (CRISPR)-associated systems
(Cas) are efficient tools for targeting specific genes for laboratory research, agricultural …

Ribonucleoprotein (RNP) complex–mediated base editing is expected to be greatly
beneficial because of its reduced off-target effects compared to plasmid-or viral vector …

Prime editing technology is capable of generating targeted insertions, deletions, and base
conversions. However, the process of designing prime editing guide RNAs (pegRNAs) …

Prime editing is a versatile and precise genome editing technique that can directly copy
desired genetic modifications into target DNA sites without the need for donor DNA. This …

Recessive dystrophic epidermolysis bullosa (RDEB) is a severe skin fragility disorder
caused by loss-of-function mutations in the COL7A1 gene, which encodes type VII collagen …

The steady progress in genome editing, especially genome editing based on the use of
clustered regularly interspaced short palindromic repeats (CRISPR) and programmable …

The mitochondrial genome has been difficult to manipulate because it is shielded by the
organelle double membranes, preventing efficient nucleic acid entry. Moreover …