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 ...

Gene editing has emerged as a powerful approach for targeting the genetic causes of disease, but getting the editing ...

Patients in a Phase I/II clinical trial conducted by UMass Chan Medical School of a dual vector gene therapy for GM2 gangliosidosis, which includes Tay-Sachs and Sandhoff diseases, exhibited a ...

Progranulin is an obvious target for a frontotemporal dementia therapy, as up to 15 percent of cases arise from loss-of-function mutations in its gene, GRN. In a study published January 7 in Science ...