A boost for the precision of genome editing
August 20, 2025
Researchers develop a fast-acting, cell-permeable protein system to control CRISPR-Cas9, reducing off-target effects and advancing gene therapy.
Researchers develop a fast-acting, cell-permeable protein system to control CRISPR-Cas9, reducing off-target effects and advancing gene therapy.
By analyzing millions of messenger RNA molecules (mRNA) during the course of ALS, researchers at Stockholm University, in collaboration with scientists at the Paris Brain Institute and Örebro University, have identified why certain nerve cells are resistant to the disease and what happens in the sensitive nerve cells when they are affected.
A study led by Roswell Park Comprehensive Cancer Center helps explain why a rare and hyper-aggressive subtype of kidney cancer is susceptible to immunotherapy—information that helped researchers create a first-of-its-kind tool to guide treatment decisions for advanced kidney cancers.
Uppsala University Hospital-led investigators report that gene-edited donor islet cells survived 12 weeks inside a man with long-standing type 1 diabetes without any immunosuppressive medication.
Amyotrophic lateral sclerosis (ALS), known as Lou Gehrig’s disease, is an incurable neurological disorder affecting motor neurons—nerve cells in the brain and spinal cord that control voluntary muscle movement and breathing.
University of North Carolina Lineberger Comprehensive Cancer Center researchers have developed a “two-in-one” molecule that can simultaneously turn off two notoriously difficult-to-target cancer-related genes, KRAS and MYC, as well as directly deliver drugs to tumors that express these genes. This advance holds special promise for treating cancers that have been historically challenging to treat.
Biomedical and genetic engineers at Duke University and the Albert Einstein College of Medicine have developed a technique that naturally increases the presence of a light-sensitive molecule throughout the body.
Heart attacks remain a leading cause of death and disability worldwide. The permanent loss of heart muscle cells—known as cardiomyocytes—and the heart’s limited regenerative capacity often lead to chronic heart failure. Current treatment strategies manage symptoms but do not repair the underlying damage.
In a first-of-its-kind clinical trial, UCLA scientists have shown it’s possible to reprogram a patient’s blood-forming stem cells to generate a continuous supply of functional T cells, the immune system’s most powerful cancer-killing agents.
Animals that hibernate are incredibly resilient. They can spend months without food or water, muscles refusing to atrophy, body temperature dropping to near freezing as their metabolism and brain activity slow to a crawl. When they emerge from hibernation, they recover from dangerous health changes similar to those seen in type 2 diabetes, Alzheimer’s disease, and stroke.