In a recent publication in Molecular Therapy, researchers from Drexel University College of Medicine and UMass Chan Medical School presented a silence-and-replace gene therapy strategy aiming to address both the gain-of-toxicity and loss-of-function components of the disease hereditary spastic paraplegia (HSP).

SYNGAP1-related disorders (SRDs) are rare neurodevelopmental conditions characterized by a wide range of symptoms, including intellectual disability, epilepsy, motor deficits and increased risk-taking behavior.

Gene editing technologies are moving forward in preclinical development with innovative strategies designed to treat diseases at their root and even reverse them.