Cell and gene therapies, or CGT, have come a long way since they were first introduced. In the last few decades, both cell therapy—the transplantation of living cells—and gene therapy—the use of ...

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.

For years, cell and gene therapies were mainly associated with rare and orphan diseases, those that impact small patient groups and lack effective treatment options. However, recent developments in ...

In the 35 years since the first gene-therapy trial for a rare genetic disease, the field has advanced to new techniques and to more diseases. That first patient, 4-year-old Ashanthi DeSilva, largely ...

Researchers in the lab of Hans-Peter Kiem, MD, PhD, at Fred Hutch Cancer Center have devised a method that could one day treat genetic hematologic disorders by correcting how the body makes blood ...

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, ...

A single one-time gene therapy could free patients with α-thalassemia, a rare and debilitating blood disorder, from the burden of lifelong transfusions. A single one-time gene therapy could free ...

This story is the first in an occasional series following Louisiana’s first patients to receive gene therapy for sickle cell disease. On the third floor of Manning Family Children’s Hospital in New ...