Circulating neurofilaments to track dorsal root ganglion toxicity risks with AAV-mediated gene therapy

Michelle A Farrar; Ewout Groen; Christiano R R Alves

doi:10.1016/j.omtm.2022.06.005

Circulating neurofilaments to track dorsal root ganglion toxicity risks with AAV-mediated gene therapy

Mol Ther Methods Clin Dev. 2022 Jun 20:26:96-97. doi: 10.1016/j.omtm.2022.06.005. eCollection 2022 Sep 8.

Authors

Michelle A Farrar^{1

2}, Ewout Groen³, Christiano R R Alves^{4

5}

Affiliations

¹ Department of Neurology, Sydney Children's Hospital Network, Level 8, Bright Alliance Building, Avoca Street, Randwick, NSW 2031, Australia.
² Discipline of Paediatrics and Child Health, School of Clinical Medicine, UNSW Medicine and Health, UNSW Sydney, Kensington, NSW 2052, Australia.
³ Department of Neurology, UMC Utrecht Brain Center, 3584 CX Utrecht, the Netherlands.
⁴ Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA 02114, USA.
⁵ Center for Genomic Medicine, Simches Research Center, Massachusetts General Hospital, Boston, MA 02114, USA.

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.

Keywords: adeno-associated virus; dorsal root ganglion; gene therapy; neurofilament; toxicity.

Publication types

News