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

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

Gene therapy represents one of medicine’s most ambitious attempts to treat disease at its root cause by altering the genetic code itself. The approach works by recovering the functions of critical ...

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

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 ...

As newborn screening and rapid DNA sequencing become routine, we are poised to catch and treat inherited diseases at their earliest stages. Today, we can intervene in the first days or weeks of life.

SEATTLE — Some of our most challenging health conditions result from genetic disorders like sickle cell disease and certain cancers. Gene therapy is revolutionizing how they're treated, and the Fred ...

After a series of deaths in patients taking Sarepta Therapeutics’ gene therapies, doubt has crept into investor sentiments ...

Onasemnogene abeparvovec gene therapy post-nusinersen or risdiplam showed meaningful motor improvements in children with SMA, with a manageable safety profile. The study cohort included older, heavier ...

Gazi University in Ankara has officially launched the production process for Türkiye's first local and national gene therapy research product. The project, which targets rare genetic diseases, is ...

Qatar set to be among top five best countries in precision health revolutionLifestyle, behaviour changes can help ‘turn on’ g ...

Despite its strong influence, APOE is not the sole cause of Alzheimer’s or other dementias. Even among people with the ...