The CRISPR (clustered regularly interspaced short palindromic repeat)/Cas9 adaptive
immunity system has been harnessed for genome editing applications across eukaryotic …

In utero gene editing has the potential to prenatally treat genetic diseases that result in
significant morbidity and mortality before or shortly after birth. We assessed the viral vector …

Abstract β-Thalassemia is a global health issue, caused by mutations in the HBB gene.
Among these mutations, HBB− 28 (A> G) mutations is one of the three most common …

CRISPR base editing is a powerful method to engineer bacterial genomes. However, it
restricts editing to single-nucleotide substitutions. Here, to address this challenge, we adapt …

Base editing is a novel genome editing strategy that enables irreversible base conversion at
target loci without the need for double stranded break induction or homology‐directed …

Programmable nucleases can introduce precise changes to genomic DNA through
homology-directed repair (HDR). Unfortunately, HDR is largely restricted to mitotic cells, and …

Most genetic diseases arise from recessive point mutations that require correction, rather
than disruption, of the pathogenic allele to benefit patients. Base editing has the potential to …

A major challenge in genetics is to identify genetic variants driving natural phenotypic
variation. However, current methods of genetic mapping have limited resolution. To address …

CRISPR-Cas has revolutionized genetic modification with its comparative simplicity and
accuracy, and it can be used even at the genomic level. Microalgae are excellent feedstocks …

Background: Mutations in low-density lipoprotein (LDL) receptor (LDLR) are one of the main
causes of familial hypercholesterolemia, which induces atherosclerosis and has a high …