Within six months, the team at Children’s Hospital of Philadelphia and Penn Medicine, along with their partners, created a therapy designed to correct KJ’s faulty gene. They used CRISPR, the ...

KJ Muldoon, a 10-month-old baby who sparked nationwide headlines after receiving a first-of-its kind gene-editing treatment, was released from the hospital this week. KJ has spent the majority of ...

While the room was abuzz with excitement that day, “he slept through the entire thing,” recalled study author Dr. Rebecca Ahrens-Nicklas, a gene therapy expert at CHOP.

Gene therapy for sickle cell disease may help improve a major contributing factor to stroke risk in patients, reports a new study from St. Jude Children's Research Hospital. Many people with ...

KJ’s gene therapy is based on CRISPR, a targeted gene-editing system which is being developed to treat cancers and a wide variety of genetic diseases. This child got a molecular pencil version ...

Some were able to detect the sounds of speech within just a few weeks. This research marks the first time gene therapy has been shown to restore hearing in both adolescents and adults.

At 12 months of follow-up gene therapy recipients with their implant turned off performed better than those who had a unilateral cochlear implant but no gene therapy when it came to distinguishing ...

The world's first patient to be treated with personalized gene editing therapy is finally headed home after over 300 days in the hospital. Nine-month-old KJ Muldoon recently underwent CRISPR-based ...

The latest fatality was a 51-year-old man who received SRP-9004, an experimental gene therapy for limb-girdle muscular dystrophy 2D/R3. All three patients died after developing acute liver failure.

The gene therapy trial was expected to complete dosing in the middle of this year. With the clinical hold in place, Shah said the timeline is uncertain. The study’s targeted enrollment was 12 ...

In the spring of 2024, the US Food and Drug Administration (FDA) approved fidanacogene elaparvovec (Beqvez), only the second gene therapy product for hemophilia B. Just a few months later, the ...