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Circulating neurofilaments to track dorsal root ganglion toxicity risks with AAV-mediated gene therapy
Michelle A Farrar1,2, Ewout Groen3, Christiano R R Alves4,5
1Department of Neurology, Sydney Children's Hospital Network, Level 8, Bright Alliance Building, Avoca Street, Randwick, NSW 2031, Australia.
Molecular Therapy. Methods & Clinical Development
|July 7, 2022
Abstract:
Identifying non-invasive biomarkers is critical to evaluate the long-term safety of adeno-associated virus (AAV)-mediated therapies. Plasma neurofilament light chain (Nf-L) levels are associated with dorsal root ganglia toxicity in rats and monkeys, suggesting that circulating Nf-L is a promising tool to be included in clinical trials and practice.
