A patient death in a CRISPR trial halts gene editing studies, raising critical safety questions. What this means for the ...

A retired HGV driver was the first person in the world to take part in a clinical trial to extend the lifespan of blood ...

A preclinical study uncovered a new gene therapy that targets pain centers in the brain while eliminating the risk of ...

A preclinical study uncovered a new gene therapy that targets pain centers in the brain while eliminating the risk of ...

Gene therapies for DMD offer potential benefits but are not curative and carry risks, necessitating informed patient and caregiver perspectives. Early diagnosis through newborn screening raises ...

Expected rapid advances in CAR T and other cellular therapies may expand treatment options not only in cancer but also in ...

Ryuji Morizane, MD, PhD, of the Department of Medicine at Massachusetts General Hospital, is the senior/corresponding author of a new paper published in Signal Transduction and Targeted Therapy, "AAV ...

The year 2023 marked a significant milestone for the treatment of sickle cell disease (SCD), with the approval of two gene therapies shown to help reduce the pain experienced by people with this ...

A new CRISPR approach can control genes without cutting DNA, opening a safer path for treating genetic diseases. A newly ...

A new CRISPR breakthrough shows scientists can turn genes back on without cutting DNA, by removing chemical tags that act ...

Gene therapy ‘switch’ may offer non-addictive pain relief. New approach targets pain signals while leaving the rest of the brain untouched.

A new gene therapy can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the amount of blood the heart can pump and dramatically improves ...