Using gene therapy, U.S. researchers have created chimeric antigen receptor (CAR)-engineered hematopoietic stem/progenitor cells (HSPCs) that have shown potential to treat HIV and other infections that can re-emerge after many years of suppression or dormancy. The CAR-engineered HSPCs were successfully engrafted in the bone marrow of pigtail macaques infected with…

A new, freely available AI catalog has classified the potential effects of millions of missense genetic mutations, which could help establish the cause of diseases such as cystic fibrosis, sickle-cell anemia, and cancer. The AlphaMissense resource from Google DeepMind categorized 89% of all 71 million possible missense variants. This compares…

A novel test that shapes pathogen nucleic acid into nanoballs could provide a cheap, sensitive way to detect such infections without the need for laboratory analysis, making it particularly useful for low-resource settings. The self-assembling nanoballs pass through a simple electrical detection system, where they disrupt the current to identify…

William A. Haseltine, PhD Hepatitis C is a serious disease that affects more than 58 million people globally.1 Although endemic in many countries, it is possible to eliminate the disease within entire populations. Unfortunately, this has only been done in a limited number of countries. Here…

A new report from a study spanning from 2000 to 2019, published Thursday in The Lancet, shows a familiar pattern of racial and ethnic group mortality gaps in the U.S. across 19 different causes of death. The study, examined data from the U.S. National Vital Statistics system and population data…

Wellcome and the Bill & Melinda Gates Foundation announced Wednesday they will provide a combined $550 million to fund a Phase III clinical trial of the tuberculosis (TB) candidate M72/AS01E (M72). If proven effective in the clinical trial, M72 could become the first new vaccine in more than 100 years…

Heligenics has launched a precision test to select among three commonly used drugs for HER2 positive breast cancer tumors: Tykerb (GSK’s laptinab), Nerlynx (Puma Biotechnology’s neratinib), and Tukysa (Seagen’s tucatinib). The test is based on Heligenic’s proprietary GigaAssay technology, which “produces mutation/function maps of all mutations,” the company reports. The…

Following up on their progress with the targeted amyotrophic lateral sclerosis (ALS) treatment Qalsody (tofersen), Biogen has made an agreement with NeuroSense Therapeutics that reinforces a biomarker-driven path. Biogen will help evaluate the effect of NeuroSense’s PrimeC on neurofilament levels in the plasma of participants in PARADIGM—a Phase IIb clinical…

A little over a decade ago, a paper was published in Science1  that made science fiction a reality. Emmanuel Charpentier, Jennifer Doudna, and colleagues reported that they had identified a means of harnessing an element of a bacterial immune system to carry out genome editing in a way that was…

Research led by the University of Pennsylvania has discovered that a viral method of entering cells can be harnessed to get therapeutic CRISPR-Cas gene editing complexes into cells more easily. Writing in the journal Nature Biotechnology, the investigators report that their new system —Peptide-Assisted Genome Editing (PAGE)—allows quick and effective…

William Haseltine With every possible combination of salt, fat, and sugar lining our grocery stores and filling fast-food restaurants, the temptation to eat junk food often feels too hard to resist. These foods tap into our deep carnal desire for pleasure. Think about the last time…

Sponsored content brought to you by As the use of next-generation sequencing (NGS) increases in the life sciences industry, and the practice is further adopted in healthcare for personalized medicine, data generation is growing at an accelerated rate. NGS has the ability to create massive data sets. With each analysis…

Vaccination against HIV-1 and Zika virus with self-amplifying replicon (rep) RNA during pregnancy can result in antibodies that are transferred to offspring before birth, preclinical research suggests. The study, outlined in the journal Molecular Therapy, showed that repRNA vaccines induced antibody responses in pregnant rabbits that were passed on in…

RNA therapeutics that will switch on in response to different physiological cues can be produced by adding a sensor that can respond to the cue as needed, according to research from the Wyss Institute at Harvard University and MIT. Using a “sense-and-respond circuit” they call detection and amplification of RNA…

An intriguing study suggests altering the balance of gut microbes could improve children’s health, particularly for those in low-income countries. Changing gut microbe composition appeared to influence growth, a key measure of health, among children living in rural Zimbabwe according to the findings in Nature Communications. The gut microbiome was…