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 ...

For patients with rare and devastating neurological disorders, treatment options can seem few and far between. However, gene therapy research, led by Dr. Russell Lonser and Dr. Brad Elder, is hoping ...

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.

A gene therapy that patients breathe in has been found to shrink lung tumours by inserting immune-boosting genes into ...

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 ...

UCLA researchers have developed a lipid nanoparticle-based gene-editing approach capable of inserting an entire healthy gene ...

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 ...

PDE6A-associated RP, a rare form of inherited retinal disease (IRD), is characterized by nyctalopia, visual field defects, and significant loss of visual acuity (VA). The disease primarily affects the ...