Research teams have created a versatile set of gene delivery systems that can reach different neural cell types in the human brain and spinal cord with exceptional accuracy. These delivery systems are ...

Researchers have released TripletDGC, a publicly available resource that links nearly 10,000 disease-associated genes to the specific cell types they most strongly affect, filling a critical gap in ...

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 ...

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 ...

Will Shrank, MD, in December 2025 unveiled a new plan for managing the high costs of an expected future wave of gene-based treatments as many drugmakers seek to more broadly apply the kind of ...