Gene therapy to target new lung cells

A map of the cells in the lungs has revealed a new cell type, the ionocyte, that plays an apparently central role in the incurable disease of cystic fibrosis. Scientists now hope that gene therapy aimed at ionocytes might revolutionise treatment of the di

NANOPARTICLES TRACK DOWN LUNG CELLS

1 Gene therapy against cystic fibrosis could include the CRISPR gene tool inserted into nanoparticles that the patient inhales. The particles end up in lung slime, where they have access to the new ionocyte cell type. The particles can be equipped with antibodies that ensure close contact with the ionocytes.

PARTICLES RELEASE GENE TOOL

2 When the antibodies on the nanoparticle bind to the ionocyte’s surface, the particle merges with the cell, so the particle’s contents end up in the cell. The contents are made up of CRISPR and a healthy version of the cell’s sick CFTR gene.

GENE TOOL REMOVES SICK GENE

3 CRISPR includes an RNA strand that tracks down the sick CFTR gene in the cell nucleus, and an enzyme that cuts up the gene. The cell tries to glue the ends together, but instead it mistakenly inserts the healthy version of the gene in the hole. So the cell now has only a healthy CFTR gene.

Cystic fibrosis causes slime to accumulate in the lungs, potentially impeding breathing.