Absence of functional deficits in rats following systemic administration of an AAV9 vector despite moderate peripheral nerve and dorsal root ganglia findings: a clinically silent peripheral neuropathy

Cheryl Tyszkiewicz, Seo-Kyoung Hwang, Jamie K. DaSilva, Ramesh C. Kovi, Kelly A. Fader, Madhu P. Sirivelu, June Liu, Chris Somps, Jon Cook, Chang-Ning Liu, Helen Wang, Absence of functional deficits in rats following systemic administration of an AAV9 vector despite moderate peripheral nerve and dorsal root ganglia findings: a clinically silent peripheral neuropathy, NeuroToxicology, 2024, ISSN 0161-813X, doi:10.1016/j.neuro.2024.02.001. 

 These findings demonstrate that there is no detectable functional consequence to the minimal-to-moderate neurodegeneration observed with our AAV9 vector treatment in rats, suggesting a functional tolerance or reserve for loss of DRG neurons after systemic administration of AAV9 vector.

Absence of functional deficits in rats following systemic administration of an AAV9 vector despite moderate peripheral nerve and dorsal root ganglia findings: a clinically silent peripheral neuropathy