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A Validatable Droplet Digital Polymerase Chain Reaction Assay for the Detection of Adeno-Associated Viral Vectors in Bioshedding Studies of Tears
Published on: July 14, 2023
Future applications: gene therapy
R M Richardson1, V Varenika, J R Forsayeth
1Laboratory for Molecular Therapeutics, Department of Neurological Surgery, University of California San Francisco, 1855 Folsom Street, Room 226, San Francisco, CA 94103, USA. richardsonma@neurosurg.ucsf.edu
Abstract:
Gene therapy for brain disorders is one of the most promising frontiers in the practice of restorative neurosurgery. There are significant experimental gene therapy initiatives underway that have led to currently active clinical trials using direct intracerebral delivery of viral vectors, and these treatments have been reported as safe and well tolerated. In the future, other clinical trials will likely use viral vectors to transfer genes that bestow on recipient tissue a desired enzymatic or neurotrophic activity relevant to the treatment of other neurodegenerative diseases, stroke, and traumatic brain injury.
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