Genetic drugs based on nucleic acid biomolecules are a rapidly emerging class of
medicines that directly reprogramme the central dogma of biology to prevent and treat …

In vivo gene editing therapies offer the potential to treat the root causes of many genetic
diseases. Realizing the promise of therapeutic in vivo gene editing requires the ability to …

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 …

Methods to deliver gene editing agents in vivo as ribonucleoproteins could offer safety
advantages over nucleic acid delivery approaches. We report the development and …

The expanding applications of nonviral genomic medicines in the lung remain restricted by
delivery challenges. Here, leveraging a high-throughput platform, we synthesize and screen …

CRISPR-Cas9 gene editing has emerged as a powerful therapeutic technology, but the lack
of safe and efficient in vivo delivery systems, especially for tissue-specific vectors, limits its …

Recently, chimeric antigen receptor (CAR)-T cell therapy has shown great promise in
treating haematological malignancies,,,,,–. However, CAR-T cell therapy currently has …

Gene-editing technologies, which include the CRISPR–Cas nucleases 1, 2, 3 and CRISPR
base editors 4, 5, have the potential to permanently modify disease-causing genes in …

Base and prime editors (BEs and PEs) may provide more precise genetic engineering than
nuclease-based approaches because they bypass the dependence on DNA double-strand …