CHOP Researchers Use Base Editing in Preclinical Model to Correct Lethal Lysosomal Storage Disease Before Birth

Adding to the growing body of literature demonstrating the feasibility of correcting lethal genetic diseases before birth, researchers at Children’s Hospital of Philadelphia (CHOP) have used DNA base editing in a prenatal mouse model to correct a lysosomal storage disease known as Hurler syndrome. Using an adenine base editor delivered in an adeno-associated viral vector, the researchers corrected the single base mutation responsible for the condition, which begins before birth and affects multiple organs, with the potential to cause death in childhood if untreated.

Fetal Surgery for Spina Bifida Leads to Better Mobility in School-Age Children

Adding to a growing body of research affirming the benefits of fetal surgery for spina bifida, new findings show prenatal repair of the spinal column confers physical gains that extend into childhood. The researchers found that children who had undergone fetal surgery for myelomeningocele, the most severe form of spina bifida, were more likely than those who received postnatal repair to walk independently, go up and down stairs, and perform self-care tasks like using a fork, washing hands and brushing teeth. They also had stronger leg muscles and walked faster than children who had their spina bifida surgery after birth.