Structural variation, such as deletions, duplications, inversions and complex
rearrangements, can have profound effects on gene expression, genome stability …

Cystic fibrosis (CF) is a genetic disease caused by mutations in the CF transmembrane
conductance regulator (CFTR) gene. The 2789+ 5G> A CFTR mutation is a quite frequent …

Studying the functional consequences of structural variants (SVs) in mammalian genomes is
challenging because (i) SVs arise much less commonly than single-nucleotide variants or …

We lack tools to edit DNA sequences at scales necessary to study 99% of the human
genome that is noncoding. To address this gap, we applied CRISPR prime editing to insert …

A 'genomically'humanized animal stably maintains and functionally expresses the genes on
human chromosome fragment (hCF;< 24 Mb) loaded onto mouse artificial chromosome …

Advancements in gene and cell therapy have resulted in novel therapeutics for diseases
previously considered incurable or challenging to treat. Among the various contributing …

Clustered regularly interspaced short palindromic repeats (CRISPR) and the associated
protein (Cas) gene editing can induce P53 activation, large genome fragment deletions, and …

The gene editing field has advanced rapidly since the development of the clustered
regularly interspaced short palindromic repeats (CRISPR)/Cas9 system because of its …

CRISPR-Cas technology is a widely utilized gene-editing tool that involves gRNA-guided
sequence recognition and Cas nuclease-mediated cleavage. The design and evaluation of …

The use of a novel and powerful technology that allows for the precise editing of the genetic
material of various organisms is becoming widespread. This technology derives from …