Researchers from the group of Hans Clevers (Hubrecht Institute) corrected mutations that cause cystic fibrosis in cultured human stem cells. In collaboration with the UMC Utrecht and Oncode Institute, they used a technique called prime editing to replace the ‘faulty’ piece of DNA with a healthy piece. The study, published in Life Science Alliance on August 9 shows that prime editing is safer than the conventional CRISPR/Cas9 technique. “We have for the first time demonstrated that this technique really works and can be safely applied in human stem cells to correct cystic fibrosis.”

Cystic fibrosis (CF) is one of the most prevalent genetic diseases worldwide and has grave consequences for the patient. The mucus in the lungs, throat and intestines is sticky and thick, which causes blockages in organs. Although treatments are available to dilute the mucus and prevent inflammations, CF is not yet curable. However, a new study from the group of Hans Clevers (Hubrecht Institute) in collaboration with the UMC Utrecht and Oncode Institute offers new hope.

Correcting CF mutations

The researchers succeeded in correcting the mutations that cause CF in human intestinal organoids. These organoids, also called mini-organs, are tiny 3D structures that mimic the intestinal function of patients with CF. They were previously developed by the same research group from stem cells of patients with CF and stored in a biobank in Utrecht. For the study, published in Life Science Alliance, a technique named prime editing was used to replace the piece of mutated DNA that causes CF with a healthy piece of DNA in these organoids.

ADS Codex translates binary data into nucleotides that can be sequenced in molecules as files for later retrieval, bringing potential cost savings and compact ‘cold storage.’

In support of a major collaborative project to store massive amounts of data in DNA

DNA, or deoxyribonucleic acid, is a molecule composed of two long strands of nucleotides that coil around each other to form a double helix. It is the hereditary material in humans and almost all other organisms that carries genetic instructions for development, functioning, growth, and reproduction. Nearly every cell in a person’s body has the same DNA. Most DNA is located in the cell nucleus (where it is called nuclear DNA), but a small amount of DNA can also be found in the mitochondria (where it is called mitochondrial DNA or mtDNA).

Through a process known as RNA interference (RNAi), scientists have been able to modify the genetic make-up of the daddy long-legs arachnid so that its distinctive spindly limbs become twice as short.

This process – which uses a gene’s own DNA sequence and small fragments of RNA to turn the gene off – was applied to the Phalangium opilio species, one of the most common species of daddy long-legs in the world.

The result is effectively a daddy short-legs instead of a daddy long-legs. The team behind the work is hoping that the experiments can teach us more about how these elongated limbs evolved in the first place.

Gene-editing technique CRISPR may deliver new treatments for genetic diseases—and it’s already being tested on patients.

17:22 minutes.

In one of the first clinical applications of the technique, last month researchers reported in the New England Journal of Medicine that CRISPR had stopped a genetic disease called amyloidosis, which occurs when an abnormal protein accumulates in your organs. They’re not the only group moving toward using CRISPR on humans; recently, the FDA approved a human clinical trial that will use the technique to edit genes responsible for sickle cell disease.