The therapeutic use of messenger RNA (mRNA) has fueled great hope to combat a wide
range of incurable diseases. Recent rapid advances in biotechnology and molecular …

Due to the fact that mRNA technology allows the production of diverse vaccines and
treatments in a shorter time frame and with reduced expense compared to conventional …

Based on engineered or bacterial nucleases, the development of genome editing
technologies has opened up the possibility of directly targeting and modifying genomic …

CRISPR–Cas9 is poised to become the gene editing tool of choice in clinical contexts. Thus
far, exploration of Cas9-induced genetic alterations has been limited to the immediate …

Genome editing holds promise for correcting pathogenic mutations. However, it is difficult to
determine off-target effects of editing due to single-nucleotide polymorphism in individuals …

Following Cas9 cleavage, DNA repair without a donor template is generally considered
stochastic, heterogeneous and impractical beyond gene disruption. Here, we show that …

Base editors use DNA-modifying enzymes targeted with a catalytically impaired CRISPR
protein to precisely install point mutations. Here, we develop phage-assisted continuous …

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 …

Decades of work have aimed to genetically reprogram T cells for therapeutic purposes,
using recombinant viral vectors, which do not target transgenes to specific genomic sites …

Versatile and precise genome modifications are needed to create a wider range of adoptive
cellular therapies,,,–. Here we report two improvements that increase the efficiency of …