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Engineered Base Editor Corrects ACTA2 Mutation in Rare Childhood Disease

Multisystemic smooth muscle dysfunction syndrome (MSMDS) is a rare condition associated with stroke, aortic dissection (tearing) and death in childhood.

13 Sep 2025

DNA-Wrapped Nanoparticles Enhance Efficiency of Gene Editing Tools

With the power to rewrite the genetic code underlying countless diseases, CRISPR holds immense promise to revolutionize medicine.

2 Sep 2025

Missing Microexons Disrupt Synapse-Related Proteins in Pediatric Glioma

A new study, led by researchers at Children's Hospital of Philadelphia (CHOP), identified tiny pieces of messenger RNA that are missing in pediatric high-grade glioma tumors but not in normal brain tissues.

19 Aug 2025

Prime Editing Reverses Symptoms of Severe Childhood Neurological Disease

Scientists use a precise form of gene editing called prime editing to correct the most common genetic mutations that cause alternating hemiplegia of childhood, a rare and severe neurological disorder that begins in infancy.

From The Jackson Labs 6 Aug 2025

UCLA Researchers Reprogram Stem Cells to Produce Continuous Cancer-Fighting T Cells

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.

5 Aug 2025

mRNA Vaccine Supercharges Immunotherapy Against Cancer

An experimental mRNA vaccine improved the tumor-fighting benefits of immunotherapy in a mouse model study, bringing researchers one step closer to their aim of generating a universal vaccination to “wake up” the immune system against cancer.

21 Jul 2025

Microglia Replacement Halts Progression of Fatal Neurological Disease in Mice and Humans

Adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP) is a progressive neurological disease with an average age of onset of 43 years and an average life expectancy of only 3 to 5 years after symptoms begin.

11 Jul 2025

New Cellular Signature Reveals Why Some Prostate Cancers Resist Standard Treatment

A new study from University of Michigan Rogel Cancer Center researchers identifies a cellular signature that explains why about one-third of prostate cancers respond especially poorly to treatment.

9 Jul 2025

Evidence for Neural Stem Cell-Based Therapies in Multiple Sclerosis

Research indicates that neural stem cell transplants could promote myelin repair in progressive multiple sclerosis, paving the way for innovative treatments.

8 Jul 2025

New Study Reveals Varied CAR T-Cell Therapy Benefits in Large B-Cell Lymphoma

A study identifies three patient subgroups in large B-cell lymphoma, revealing distinct responses to CD19 CAR T cell therapy and enhancing precision medicine.

20 Jun 2025

Researchers Develop Promising Vaccine for Vaginal Yeast Infections

The UGA vaccine offers a potential breakthrough in combating vaginal yeast infections, aiming to reduce reliance on antifungal drugs amid growing resistance.

18 Jun 2025

TUM Scientists Develop Breakthrough Method to Monitor CAR-T Cells

In modern immunotherapy, modified immune cells are introduced into the body to attack tumors and other targets.

16 Jun 2025

Proteomic Signature in Blood Predicts Dementia and Diabetes Risk

A blood-based proteomic signature predicts healthspan, linking protein levels to disease risk and mortality, paving the way for targeted aging interventions.

10 Jun 2025

Ultra-Rapid Genetic Test Revolutionizes Brain Tumor Diagnosis

Scientists and medics have developed an ultra-rapid method of genetically diagnosing brain tumors that will cut the time it takes to classify them from 6-8 weeks, to as little as two hours – which could improve care for thousands of patients each year in the UK.

21 May 2025