Life Biosciences is pushing cellular rejuvenation into the clinic with ER-100, an experimental gene ...

Gene-editing tools like CRISPR have unlocked new treatments for previously uncurable diseases. Now, researchers at the ...

A new CRISPR-based one-off procedure that lowers "bad" cholesterol has been approved to enter Phase I human trial. If ...

Fifteen-year-old Jayden Wilsey of Falmouth is the first person at Boston Children's Hospital to receive human gene therapy for sickle cell disease, following approval of the therapy in 2024 by the U.S ...

Two-year follow-up data demonstrate sustained benefit, including improvements in pain, fatigue and bone health, following single low dose of ...

A new study has brought scientists one step further in the direction of developing a cure for a brutal group of rare brain disorders known as SYNGAP1-related disorders, or SRDs. Researchers were able ...

Gene therapy holds the promise of preventing and curing disease by manipulating gene expression within a patient's cells. However, to be effective, the new gene must make it into a cell's nucleus. The ...

NHS Scotland has approved a one-time CRISPR gene therapy for severe sickle cell disease, offering durable reduction in vaso-occlusive crises for eligible patients.

Biopharmaceutical company Medera Inc. said it completed patient enrollment for Cohort B of its clinical trial evaluating SRD-002 gene therapy for heart failure with preserved ejection fraction (HFpEF) ...

Future GLP-1 gene therapy could enable long-term hormone production, with Fractyl Health and others planning human trials in 2024.

Scientists achieved a genetic breakthrough by removing an extra human chromosome using gene-editing technology. This landmark study successfully deleted the surplus chromosome 21 responsible for Down ...