Gene therapy holds the promise of preventing and curing disease by manipulating gene expression within a patient's cells.

Precision DNA editing gene therapy achieves deep remissions in aggressive leukemia previously considered incurable.

After facing a delayed decision deadline and a clinical hold, Regenxbio’s Hunter syndrome gene therapy has been rejected by ...

Delivery of therapeutic genes is essential for gene therapy. Adeno-associated viruses (AAVs) are a prime vector for carrying ...

A research team has successfully demonstrated the world's first gene-editing treatment for Leber's hereditary optic ...

Gene-editing tools like CRISPR have unlocked new treatments for previously uncurable diseases. Now, researchers at the ...

Fixing gene defects with designer DNA is on the horizon after Google DeepMind launched a pioneering new AI tool.

DNA Nanobots, a pioneer in self-assembling nanoscale therapeutics, today announced it has secured $3.5 million in funding from a prominent, family office. This significant investment solidifies the ...

Researchers developed a topical gene therapy that corrects disease-causing mutations in human skin models using lipid ...

The disease leads to the progressive growth of fluid-filled cysts in the kidneys, often resulting in kidney failure and other ...

Gene therapy is a medical treatment that works by changing or fixing a person's genes to treat or prevent disease ...

Forbes contributors publish independent expert analyses and insights. A patient in a late-stage gene editing trial for a rare heart condition died from fatal liver complications after receiving an ...