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

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

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

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 ...

With a shared purpose, we can realize the true promise of CRISPR and improve healthcare, providing hope to more patients with ...

The special properties of methylcellulose foam could make it a vehicle for bedside genetic engineering, according to a proof-of-principle study from bioengineers at Fred Hutch Cancer Center. In the ...

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

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

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 EMA’s positive opinion on Kygevvi marks a turning point for patients with TK2d, an ultra-rare genetic muscle disease previously managed only with supportive care.