Reducing microglial activity in the presence of apolipoprotein E4 (APOE4) has uncovered a mechanism associated with the deposition of misfolded amyloid and tau in a novel mouse model of Alzheimer’s disease. By transplanting human neurons into the mouse brain and eliminating the mouse microglia, scientists at the Gladstone Institutes in San Francisco observed that amyloid and tau deposition was reduced. These results support therapeutic strategies that target APOE4 and microglia.

Researchers at Harvard Medical School have found that blocking the neuron-released peptide CGRP decreases pain sensitivity and reduces lesion size in endometriosis. Endometriosis is a painful, steroid-dependent inflammatory condition in which tissue similar to that of the endometrial lining grows and establishes outside the uterine mucosa.

Gene editing strategies, from epigenetic engineering to cell reprogramming and genetic vaccines, are accelerating the development of new therapies that awaken the immune system to treat cancer, as presented last month in Rome at the 31st Annual Congress of the European Society of Gene and Cell Therapy (ESGCT). Some of these advances are taking advantage of the conditions of the tumor microenvironment, where cancer cells coexist with immune cells, microorganisms and blood vessels.

Currently, cancer therapy trial-and-error methodology is inefficient and unsustainable. Oncology is the worst therapeutic area for drug trial success; only 3.4% of drugs that enter phase I end up being FDA approved, and 57% fail due to poor drug efficacy in trials. Building tools that may aid in predicting an individual’s response to a specific therapy may help in reducing costs, guesswork, and importantly improve the outcome of patients and accelerate new drug development.

Scientists from different laboratories around the world have presented the latest advances in research into malignant brain tumors at the 31st Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), which is being held Oct. 22 to 25 in Rome.

A new study helps explain the role of genetic variation in shaping gene regulation in the Indonesian archipelago, one of the most diverse regions in the world. “This study is the only study of splicing from Southeast Asian populations. There is basically no data from this part of the world,” study author Irene Gallego Romero told BioWorld. For drug discovery, most of the people that have historically participated in clinical trials are of European ancestry, and scientists are just beginning to study African populations to better understand genetic differences in these populations, said Romero, a population geneticist and biological anthropologist at the University of Melbourne.

David Baker, Demis Hassabis and John Jumper share the 2024 Nobel Prize in Chemistry for their contributions to the science of protein structure. David Baker was awarded half the prize “for computational protein design,” according to the Royal Swedish Academy of Sciences. Hassabis and Jumper shared the other half “for protein structure prediction.”

David Baker, Demis Hassabis and John Jumper share the 2024 Nobel Prize in Chemistry for their contributions to the science of protein structure. David Baker was awarded half the prize “for computational protein design,” according to the Royal Swedish Academy of Sciences. Hassabis and Jumper shared the other half “for protein structure prediction.”

David Baker, director of the Institute for Protein Design at the University of Washington School of Medicine, is a pioneer in protein design. His contributions have been recognized with countless awards, and now, a place among the 2024 Clarivate Citation Laureates. Baker’s lab has developed several open-source software applications for nanotechnology and biomedicine. With these methods, scientists build new proteins that bind to drug targets and block them or activate cellular signals.

Research into the regulation of gene expression experienced a significant breakthrough with the discovery of microRNA, small RNA molecules that do not code for proteins but control their translation. This finding has earned its discoverers – Victor Ambros and Gary Ruvkun – the 2024 Nobel Prize in Physiology or Medicine “for the discovery of microRNA and its role in post-transcriptional gene regulation.”