CRISPR-Cas gene editing has revolutionized experimental molecular biology over the past
decade and holds great promise for the treatment of human genetic diseases. Here we …

Organoids bridge the gap between 2D cell lines and in vivo studies. With their 3D
organization and cellular heterogeneity, adult stem cell-derived organoids closely resemble …

Prime editing recently emerged as a next-generation approach for precise genome editing.
Here we exploit DNA double-strand break (DSB) repair to develop two strategies that install …

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

Introduction Cystic fibrosis (CF), a potentially fatal genetic disease, is caused by loss-of-
function mutations in the gene encoding for the CFTR chloride/bicarbonate channel …

Breast cancer is the leading cause of cancer-related deaths in women. The aggressive
breast cancer subtype is commonly linked to the genetic alterations in the TP53 tumor …

Prime editors have high potential for disease modelling and regenerative medicine efforts
including those directed at the muscle-wasting disorder Duchenne muscular dystrophy …

Inherited retinal diseases (IRD) are a clinically and genetically heterogenous group of
diseases and a leading cause of blindness in the working-age population. Even though …

Organoids are three-dimensional, multicellular constructs that recapitulate the structural and
functional features of specific organs. Because of these characteristics, organoids have …

Prime editing is a recent, CRISPR-derived genome editing technology capable of
introducing precise nucleotide substitutions, insertions, and deletions. Here, we present …