Vertex has just filed for approval of Kalydeco for CF patients aged 18 and older who have the R117H mutation, the most common residual function mutation, which could add another 300 patients to ...

To achieve this, the donated cells must replicate again and again — and again. It’s a cycle that might put recipients at risk if a new, potentially harmful mutation arises or if a mutation present in ...

Jan. 8, 2025 — Researchers describe minimal versatile genetic perturbation technology (mvGPT). Capable of precisely editing genes, activating gene expression and repressing genes all at the same ...