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

Michelle A. Farrar | Zendy; Ewout J. N. Groen | Zendy; Christiano R. R. Alves | Zendy

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

Author(s) -

Michelle A. Farrar,

Ewout J. N. Groen,

Christiano R. R. Alves

Publication year - 2022

Publication title -

molecular therapy — methods and clinical development

Language(s) - English

Resource type - Journals

SCImago Journal Rank - 2.285

H-Index - 32

ISSN - 2329-0501

DOI - 10.1016/j.omtm.2022.06.005

Subject(s) - dorsal root ganglion , neurofilament , toxicity , dorsum , genetic enhancement , gene , anatomy , biology , medicine , neuroscience , pathology , genetics , immunohistochemistry

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.

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