Volume 22 Issue 11, November 2023

A pack of CAR T cell therapies are now in trials in systemic lupus erythematosus, as the race begins to prove the potential of autologous cell therapies in autoimmune diseases.

CRISPR-based genome editing has the potential to treat many human genetic diseases, but achieving stable, efficient and safe in vivo delivery remains a challenge. This Review assesses current delivery systems for genome editors—focusing on adeno-associated viruses and lipid nanoparticles—and highlights data from recent clinical trials. Emerging delivery systems and ongoing challenges in the field are discussed.

Advances in computational omics technologies are enabling access to the hidden diversity of natural products, and artificial intelligence approaches are facilitating key steps in harnessing the therapeutic potential of such compounds, including biological activity prediction. This article discusses synergies between these fields to effectively identify drug candidates from the plethora of molecules produced by nature, and how to address the challenges in realizing the potential of these synergies.

Duchenne muscular dystrophy is an inherited muscle-wasting disease caused by mutations that disrupt production of dystrophin protein. This Review discusses the plethora of therapeutic approaches being developed to restore dystrophin function, such as exon skipping, gene replacement, cell therapy and gene editing, and highlights recent clinical approvals.