People with the rare inherited metabolic disorder Gaucher disease have a deficiency in the lipid-digesting glucocerebrosidase enzyme, which causes the accumulation of harmful levels of glucolipids in various organs. The enzyme has a very short half-life, which rules out enzyme replacement as an effective therapy, and as things stand, there are few treatments for this and other lysosomal storage diseases (LSDs). Now, researchers have discovered two small molecules that enhance the activity of glucocerebrosidase in cellular models of LSD, pointing to a potential new approach to treating these diseases.

Scientists at Harvard Medical School have shown that in mice lacking amyloid beta (Aβ), the fundamental hallmark of Alzheimer's disease (AD), neurons died from the effect of the most harmful mutation of this neurodegenerative disease. They showed that presenilin (PS) could be behind the origin of the disease without the need for Aβ. They maintain that it is time to update theories and redirect efforts.

A new study has shown that when a gene is mutated and its copy number is altered, the risk of that gene contributing to the development of cancer increases. Although it was already known that both variations together promoted cancer, it had not been described how the link between the two leads to the progression of tumors and what implications it had on the genetics of cancer.

The gastrointestinal tract could be key to developing new drugs to combat resistant bacteria. Computational analysis of the human microbiome has revealed a new class of peptides with antimicrobial potential that, once synthesized, inhibited the growth of several microorganisms in vitro and in vivo.

Infection or cure? Scientists from Tel Aviv University and the University of Glasgow genetically modified the Toxoplasma gondii to bring a protein inside neurons. The novelty of using a protozoan that can travel from the gut to parasitize the CNS contrasts with the possibility of causing a disease. The scientists are already working on how to avoid it.

A strategy inspired by deficient HIV replication could be used as a treatment to reduce viral load in patients living with HIV and help control the pandemic of the retrovirus. Scientists from the University of California San Francisco want to use HIV against itself by using a parasitic version of the pathogen.

Infection or cure? Scientists from Tel Aviv University and the University of Glasgow genetically modified the Toxoplasma gondii to bring a protein inside neurons. The novelty of using a protozoan that can travel from the gut to parasitize the CNS contrasts with the possibility of causing a disease. The scientists are already working on how to avoid it.

To be successful, CAR T-cells need a balance between being effective and overkill. Researchers from the University of Pennsylvania and Vittoria Biotherapeutics Inc. have eliminated the CD5 signaling pathway of their CAR Ts to prevent the immunosuppressive brake effect. In return, this improved their proliferation and antitumor activity in T cell lymphomas.

Increased life expectancy also comes with age-related diseases. But what causes one to age? Although there is no single answer, scientists at Duke-National University of Singapore have shown in mice that interleukin-11 (IL-11) promoted aging. Blocking it improved health and lifespan.

Not all cancer cells that detach from the primary tumor and embark on a journey to another organ colonize it. To establish themselves, cells need specific conditions. Scientists at the Federal Institute of Technology Zurich (ETH Zurich) explored this hypothesis in the liver and identified a protein involved in metastatic transformation. Blocking it prevented liver metastasis. Their findings may have applications in other organs and various types of cancers.