Key result
AAV-RNAi constructs and recombinant AAV vectors promote dorsal root ganglion (DRG) toxicity across multiple animal models.
AAV-RNAi constructs are associated with dorsal root ganglion toxicity in preclinical models.
May warrant DRG toxicity monitoring in AAV gene therapy; leaves open human translation pending further studies.
Adeno-associated virus (AAV)-based gene therapies have been approved for treating several rare genetic diseases in humans, including those related to central nervous system (CNS) disorders. Delivery of recombinant (r)AAVs to the CNS has been shown to be effective in preclinical studies and clinical trials. While rAAV vectors are generally considered safe, dorsal root ganglion (DRG) toxicity following rAAV treatment has been observed across multiple animal models, including non-human primates (NHPs), piglets, rabbits, and rats.
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Xie et al. (2024) studied Central nervous system disorders / DRG toxicity. AAV-RNAi constructs and rAAV vectors was evaluated on Dorsal root ganglion (DRG) toxicity. AAV-RNAi constructs and recombinant AAV vectors promote dorsal root ganglion (DRG) toxicity across multiple animal models.
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