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 ...

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

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 ...

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 ...

Recent therapies like hydroxyurea (Droxia, Bristol-Myers Squibb), the first FDA-approved drug for SCD in 1994, increase fetal hemoglobin (HbF), which, through unknown pathways, improves SCD symptoms ...

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.

Despite groundbreaking research, many cell and gene therapies do not make it all the way to the patients. Researchers and clinicians in Lund have now presented a new model for cooperation that will ...

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 ...

After telegraphing an investigation in November, the FDA has moved to restrict the use of bluebird bio’s gene therapy Skysona in certain patients. The FDA has updated Skysona’s indication, allowing it ...

After facing a delayed decision deadline and a clinical hold, Regenxbio’s Hunter syndrome gene therapy has been rejected by ...

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 ...