Gene therapy, a technique that is revolutionizing the treatment of multiple genetic conditions, including eye and muscle diseases and blood disorders, requires efficient and specific delivery of the genetic material to the tissue and cell type of interest. To help improve gene therapy, a multidisciplinary team led by researchers at Baylor College of Medicine, the Jackson Laboratory and the University of Massachusetts Medical School has generated a comprehensive atlas that researchers can use to select the most effective viral vehicle for their target organ.

A team of scientists from The University of Texas Health Science Center at San Antonio (UT Health San Antonio) has identified a promising new drug target for acute myeloid leukemia (AML), a deadly blood cancer with a five-year survival rate of just 30%, according to the National Cancer Institute. Their study, published in Cell Stem Cell in February, highlights the crucial role of a protein called paraspeckle component 1 (PSPC1) in the progression of AML.

People with low levels of low-density lipoprotein cholesterol (LDL-C) in their blood have a lower risk of dementia, including lower risk of Alzheimer's disease related dementia, shows a study published online today in the Journal of Neurology Neurosurgery & Psychiatry.

Use of statins conveyed an additional protective effect for people with low LDL-C, specifically those with blood levels less than 1.8 mmol/L (<70 mg/dL), reducing their risk of dementia even further.

Researchers develop innovative phospholipids that improve the functional delivery of mRNA via lipid nanoparticles, paving the way for advanced therapeutic applications.

A groundbreaking study conducted by researchers at Hokkaido University has unveiled a novel class of zwitterionic phospholipids capable of significantly enhancing the functional delivery of mRNA. The study was published in the Advanced Science.

A scientific technique that rapidly increases the body's production of anti-inflammatory cells promoted healing from heart attacks in mice, according to a new study by investigators from the Smidt Heart Institute at Cedars-Sinai. Once adapted to treat humans, the technique could potentially be used to repair heart muscle damage after a heart attack and be applied to a variety of inflammatory disorders.

The investigators' findings were published in the peer-reviewed Journal of Clinical Investigation.

Scientists have long struggled to target proteins that lack defined structure and are involved in cancer, neurodegenerative disorders like Parkinson's disease, and other serious illnesses. Now, a new study from Scripps Research demonstrates a proof of concept for a new strategy: engineering proteases - enzymes that cut proteins at specific sites - to selectively degrade these elusive targets with high precision in the proteome of human cells.

Among other functions, the NK cells (Natural Killers, a type of lymphocyte forming part of the immune system) have the capacity to detect and eliminate cancer cells. But in some cases they cannot overcome the tumour’s defense mechanism and the cancer grows. Now, a study published in Nature Immunology, with the involvement of the Hospital del Mar Research Institute, the Universitat Autònoma de Barcelona and the Pompeu Fabra University, proposes a new approach to strengthen NK cells in their fight agaist tumour cells.