For adeno-associated viral gene therapies to scale, scientists must first address safety and manufacturability challenges. Improving payload design and optimizing production systems can help ...

ORLANDO -- A gene therapy that could transform CAR T-cell treatment achieved rapid and potentially durable measurable ...

Using X-ray diffraction data, James Watson (1928-2025) made the seminal discovery of the double-helical structure of DNA ...

Creating physiologically relevant conditions for cells that replicate in vivo environments can improve experimental results, ...

Rapid innovation, expanding clinical pipelines, and rising demand for curative treatments position gene therapy as a ...

Gene therapy is a revolutionary field in modern medicine, offering transformative potential to treat and potentially cure a wide range of genetic and acquired diseases. By addressing the root cause of ...

In a world-first trial, scientists used a one-off CRISPR gene edit to switch off a liver “fat brake” gene, slashing stubborn LDL cholesterol and triglycerides in patients whose levels refused to budge ...

StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models. Gene therapy can effectively treat various ...

Engineered oncolytic bacteria have emerged as a promising therapeutic platform for precision cancer treatment, offering tumor ...

Over the last 15 years, I have witnessed how cell and gene therapy (CGT) has evolved in many ways, spanning from research and development to manufacturing. Breakthroughs such as CAR-T and gene therapy ...