A single-dose gene replacement therapy is found to improve movement ability in children over 2 years of age and teenagers with spinal muscular atrophy, according to research published in Nature ...

Treating our first patient is a proud moment for Abeona and a testament to the tireless resolve of our team,” said Vish ...

Current gene therapies to treat sickle cell disease are complex, time-consuming, and are sometimes linked to serious side effects like infertility or blood cancer. To address these challenges, Johns ...

While gene therapy for cystic fibrosis is still in the research phase, researchers aim to correct the defective gene responsible for the disease. Current research is promising. Cystic fibrosis (CF) is ...

An activity pattern in certain genes responsible for building proteins known as spleen tyrosine kinases can predict which melanoma patients are likely to have severe side effects from immunotherapy ...

Delivering the hormone leptin directly to the brain through gene therapy aids weight loss without the significant side effect of bone loss, according to new collaborative research from Oregon State ...

Almost two thirds of patients with a rare form of blood cancer who took part in a clinical trial of a revolutionary new ...

Preliminary results from two trials of the gene therapy exagamglogene autotemcel (exa-cel) suggest the therapy offers an ...

This story is part of a series on the current progression in Regenerative Medicine. In 1999, I defined regenerative medicine as the collection of interventions that restore tissues and organs damaged ...

The FDA approved the intrathecal gene therapy onasemnogene abeparvovec (Itvisma) to treat spinal muscular atrophy (SMA) in ...

FDA cleared Itvisma as the first gene-replacement therapy available to people 2 and older with the rare muscle-weakening condition. ・Phase III data showed improved or stabilized motor function with a ...