A gene therapy that patients breathe in has been found to shrink lung tumours by inserting immune-boosting genes into ...
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 ...
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 ...
Explore how community blood centers are shaping the future of cell and gene therapies, from customized collections to ...
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.
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 ...
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 Pfizer ...
Alzheimer’s may be driven far more by genetics than previously thought, with one gene playing an outsized role. Researchers found that up to nine in ten cases could be linked to the APOE gene — even ...
After a series of deaths in patients taking Sarepta Therapeutics’ gene therapies, doubt has crept into investor sentiments ...
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 ...
Qatar set to be among top five best countries in precision health revolutionLifestyle, behaviour changes can help ‘turn on’ g ...
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