The first human trial of prenatal gene therapy is coming soon, aiming to treat babies with the fatal disorder GM1 gangliosidosis before birth. Scientists hope the study could open the door to treating more genetic diseases in the womb—before irreversible damage begins. https://ucsfh.org/4tVvs3e
A planned first-in-human trial aims to treat GM1 gangliosidosis in utero, possibly laying the foundation for prenatal gene therapies for other genetic diseases.
