Gene editing can repair a DNA error in mice that causes Dravet syndrome, a rare, incurable, and potentially deadly form of ...

In an oral presentation at ASGCT, Editas reported that a single dose of EDIT-401 achieved ≥90 percent mean LDL-C reduction across all dose groups in non-human primates (NHPs). ≥90 percent mean LDL-C ...

In a world first, a research team at the University of Zurich has successfully treated mice carrying an inherited form of ...

The ethics of using safe gene therapies to improve the health and cognition of Down syndrome children and adults.

Researchers breakthrough gene silencing in chickens, enabling disease-resistant poultry. Learn how this genetic engineering advance brings commercial gene-edited flocks closer to reality.

What was achieved: Researchers corrected SCN1A mutations in mice using prime or base editing, reducing seizures and improving survival. Why it matters: These are the first demonstrations of directly ...

End-to-end pipelines and regulatory advances aim to expand gene editing to broad patient populations Ajay Gannerkote, ...

A biotech company is looking to get approval next after their recent discovery.

A tiny salamander that can regrow an entire limb may have just revealed one of the biggest clues yet for future human ...

New preclinical data show PBGENE-DMD drove up to a 3x higher dystrophin protein restoration in skeletal muscle and up to 12x higher in respiratory muscle in early-juvenile mice compared to ...