Gene therapy holds the promise of preventing and curing disease by manipulating gene expression within a patient's cells. However, to be effective, the new gene must make it into a cell's nucleus. The ...

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

In the 35 years since the first gene-therapy trial for a rare genetic disease, the field has advanced to new techniques and to more diseases. That first patient, 4-year-old Ashanthi DeSilva, largely ...

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

Adeno-associated virus (AAV) has emerged as a reliable vehicle for delivering therapeutic DNA into patient cells, with eight AAV-based gene therapies approved worldwide as of mid-2025. 1 Yet questions ...

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 ...

Nine-year follow-up of gene therapy in haemophilia B confirms long-term safety and reduced bleeding, though full capsid ...

Researchers have found a promising new method for gene therapy. They successfully restarted inactive genes by bringing them closer to genetic switches on the DNA called enhancers. The intermediate ...

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 ...

Editor’s note: On June 3, 2025, KJ Muldoon was discharged from the hospital and is now at home with his family. When a baby born in Philadelphia was announced as the first person to get a gene therapy ...

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 ...

What you need to know: Gene therapy is a medical treatment that works by changing or fixing a person's genes to treat or prevent disease A biotechnology company in Uganda has developed a new ...