Genetic Engineering Publications - GEG Tech top picks

Six people with a rare and deadly disease called transthyretin amyloidosis were treated with CRISPR-Cas 9 in a clinical trial. The treatment targets a protein that is primarily made in the liver.

Preliminary results from this clinical trial suggest that CRISPR-Cas9 gene editing can be deployed directly in the body to treat the disease. This is the first study to show that the technique can be safe and effective if the CRISPR-Cas9 components are infused into the bloodstream. All patients experienced a decrease in the level of a deformed protein associated with the disease. Being able to edit genes directly in the body would open the door to treating a wider range of diseases.

An experimental malaria vaccine containing live parasites protected almost all subjects from infection in a small clinical trial.

Participants in the study received an injection containing live Plasmodium falciparum, as well as drugs to kill any parasites to kill any parasites that had reached the liver or bloodstream, where they can where they can cause malaria symptoms. The vaccine protected 87.5 percent of participants infected with the strain of parasite used for inoculation, and 77.8% of those infected with a different strain.