Cardiovascular disease is the world’s leading killer and one of the most economically damaging, as its prevalence and costs have been rising. According to our research, offering a one-time ...

Genetic disorders occur due to alterations in the primary genetic material—deoxyribonucleic acid (DNA)—of an organism.

CRISPR–Cas9-based therapies are widely investigated for their clinical applications. However, there are limitations ...

In a Phase 1, first-in-human trial, a one-time infusion of an investigational CRISPR-Cas9 therapy targeting angiopoietin-like protein 3 (ANGPTL3) was safe and reduced LDL cholesterol by nearly 50% and ...

UCSF scientists engineered old fibroblast cells to turn their genes on and off in the same way as young fibroblasts. The old ...

Instead of requiring personalized gene edits for each patient, the new approach could create a standardized method to use for many diseases. By Pam Belluck and Carl Zimmer Gene-editing therapies offer ...

Revolutions in gene therapy are rapidly changing the landscape of modern medicine. Revolutions in gene therapy are rapidly changing the landscape of modern medicine, forcing society and science alike ...

Scientists have discovered that a “longevity gene” found in people who live beyond 100 can reverse heart aging in models of Progeria, a devastating disease that causes children to age rapidly. By ...

A new gene-editing technique enables the correction of multiple genetic mutations simultaneously, transforming the prospects for millions living with complex inherited diseases, such as cystic ...

The gene therapy market is surging toward $36.55 billion by 2032, powered by rising demand for curative treatments that target genetic diseases at their source([1]) . The FDA approved three ...