CRISPR gene therapy Casgevy can nearly eliminate sickle cell crises, but difficult stem‑cell collection is delaying access ...

Gene therapies have the potential to cure some diseases, but they are extraordinarily expensive. Location can also be a big ...

In one of the biggest advances against leukemia and other blood cancers in many years, doctors are reporting unprecedented success by using gene therapy to transform patients' blood cells 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 ...

As cell and gene therapies move into more hospitals and cancer centers, health system leaders are learning that clinical success is only part of the equation. These treatments may offer life-changing ...

The Food and Drug Administration aims to evaluate treatments for rare diseases based on plausible evidence that they would work — without requiring a clinical trial first.

As Uganda rolls out mandatory nationwide screening of newborns for sickle cell disease this month, a gene therapy celebrated ...

Despite innovative new research, many cell and gene therapies do not make it all the way to the patients. Researchers and ...

Health systems learn why cell and gene therapy success depends on workflows, staffing, and finances—not just science—and how to build them.

"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 ...

Children with hereditary deafness regained their hearing thanks to a type of gene therapy, a new study published on Wednesday found. In a clinical trial, co-led by investigators from Mass Eye and Ear, ...

NANJING, China — A groundbreaking clinical trial has achieved what many thought impossible: restoring meaningful hearing in people born profoundly deaf, including teenagers and young adults who were ...