The authors conducted a Phase I trial of a gene editing approach delivered by adeno-associated virus vector carrying a codon-optimized human giant axonal neuropathy transgene. The transgene targets anterior horn motor neuron bodies and sensory neurons in the dorsal root ganglia with the goal of repairing peripheral nerves and their axons. Each patient received a single intrathecal injection of the investigational agent. At one year, patients had less progression of disease compared to pretreatment intervals.
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