The discovery of DNA was a milestone in the history of science that led to a breakthrough in biomedical research. By associating disease and genetics, genome correction techniques were ultimately developed that are supposed to work in the same way that antibiotics and antivirals block pathogenic microorganisms: by directly attacking the causes of disease.

The most ambitious objective of any treatment is to eradicate the disease, acting on its origin to cure it instead of treating its symptoms. This is the purpose of the gene therapy against type 2 diabetes (T2D) and obesity that Fractyl Health Inc. is developing. Scientists from the Lexington, Mass.-based company have designed a strategy based on glucagon-like peptide-1 (GLP-1) to transform pancreatic cells and reverse the disease.

Investigators have identified a second individual who remained cognitively normal into his late 60s despite having the PSEN1 E280A mutation, which causes a familial version of early-onset Alzheimer’s disease (AD). The likely source of protection, a mutation in a gene called Reelin, is distinct from the protective mechanism identified in the first case of an individual who was protected from the effects of PSEN1 E280A. That case was reported in 2019.