The advent of clustered regularly interspaced short palindromic repeat (CRISPR) genome
editing, coupled with advances in computing and imaging capabilities, has initiated a new …

Programmable gene-editing tools have transformed the life sciences and have shown
potential for the treatment of genetic disease. Among the CRISPR–Cas technologies that …

Mutations in the TP53 tumour suppressor gene are very frequent in cancer, and attempts to
restore the functionality of p53 in tumours as a therapeutic strategy began decades ago …

Over the past decade, CRISPR has become as much a verb as it is an acronym,
transforming biomedical research and providing entirely new approaches for dissecting all …

Cell-based therapeutics are an emerging modality with the potential to treat many currently
intractable diseases through uniquely powerful modes of action. Despite notable recent …

The targeted deletion, replacement, integration or inversion of genomic sequences could be
used to study or treat human genetic diseases, but existing methods typically require double …

Prime editing enables the installation of virtually any combination of point mutations, small
insertions or small deletions in the DNA of living cells. A prime editing guide RNA (pegRNA) …

Thalassaemia is a diverse group of genetic disorders with a worldwide distribution affecting
globin chain synthesis. The pathogenesis of thalassaemia lies in the unbalanced globin …

Cytosine base editors (CBEs) are larger and can suffer from higher off-target activity or lower
on-target editing efficiency than current adenine base editors (ABEs). To develop a CBE that …

Multiple clinical trials of allogeneic T cell therapy use site-specific nucleases to disrupt T cell
receptor (TCR) and other genes,,,,–. In this study, using single-cell RNA sequencing, we …