Scientists at St. Jude Children's Research Hospital and Northwestern University identified a previously unknown treatment opportunity for sickle cell disease and β-thalassemia. The discovery, ...

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 ...

Researchers have taken an important step toward solving one of the biggest challenges in gene therapy for children with ...

Read more: Click here to read the original, longer version of this story. AND… breathe. Twenty-six years after the gene implicated in cystic fibrosis was found, gene therapy has been able to help ...

Realizing the potential of cell and gene therapies will require organizations to rethink how they finance care and build the ...

Preliminary results from two trials of the gene therapy exagamglogene autotemcel (exa-cel) suggest the therapy offers an effective cure for beta-thalassemia and sickle cell disease in children younger ...

"This was the first proof that 'gene therapy in a box' could work." Gene therapies or cell therapies that involve genetically modified cells today are available at only a limited number of research ...

A well-connected family’s fight to access a potential treatment for their son highlights the barriers most rare disease ...

The use of CRISPR-Cas9 gene editing technology has potential to transform the field of gene therapy through its simple and cost-effective delivery mechanism, said Peter Marks, director of the US Food ...

Forbes contributors publish independent expert analyses and insights. William A. Haseltine, Ph.D., covers genomics and regenerative medicine This voice experience is generated by AI. Learn more. This ...

Duchenne muscular dystrophy (DMD) is a neuromuscular disorder that results from mutations in the DMD gene. Gene therapies for DMD change genetic material in a person’s body to treat this condition.