Central nervous system gene therapy has entered a new development paradigm. New techniques are being employed for a wide range of illness indications and pathways
Moataz Dowaidar
Abstract
As a result of recent favorable results in phase I–II studies in human patients, as well as improved vector technology, gene therapy for central nervous system (CNS) disorders has entered a new development paradigm. These approaches are currently being used for a wide range of disease indications and pathways, considerably expanding the scope of gene therapy for CNS diseases. This expansion involves both the simplicity of delivery techniques and the anticipation of higher vector production requirements, particularly for IV targeting. Standardized delivery strategies that may be adopted in a variety of locales throughout the world are needed to serve an increasing number of patients. Furthermore, manufacturability is a critical factor in the development of the GT sector, as it influences not only the creation of high-quality, large-quantity vectors to meet future clinical demand, but also the cost and our ability to make innovative gene therapy products accessible to all patients.