Base editing—the introduction of single-nucleotide variants (SNVs) into DNA or RNA in
living cells—is one of the most recent advances in the field of genome editing. As around …

Genome editing has the potential to treat an extensive range of incurable monogenic and
complex diseases. In particular, advances in sequence-specific nuclease technologies have …

To date, almost 2600 gene therapy clinical trials have been completed, are ongoing or have
been approved worldwide. Our database brings together global information on gene therapy …

The clustered, regularly interspaced, short palindromic repeats (CRISPR)/CRISPR-
associated protein (Cas) systems are efficient and versatile gene editing tools, which offer …

Host: pathogen interactions dictate the outcome of infection, yet the limitations of current
approaches leave large regions of this interface unexplored. Here, we develop a novel …

The CRISPR/Cas systems in prokaryotes such as bacteria and archaea are the adaptive
immune system to prevent infection from viruses, phages, or other foreign substances. When …

CRISPR/Cas9 is an effective gene editing tool with broad applications for the prevention or
treatment of numerous diseases. It depends on CRISPR (clustered regularly interspaced …

The concept of gene therapy arose during the 1960s and early 1970s. Rogers and Pfuderer
1 carried out the first genetic modification of a virus (Tobacco mosaic virus [TMV]), and …

Abstract Background Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)
is derived from the bacterial innate immune system and engineered as a robust gene-editing …

Severe congenital neutropenia (SCN) is a life-threatening disorder most often caused by
dominant mutations of ELANE that interfere with neutrophil maturation. We conducted a …