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

HIGHLIGHTS

• who: Michelle A. Farrar from the cellular stress and neurodegeneration, A further study reported prevention of neuronal degeneration and axonopathy when using AAV gene therapy designed to include a specific microRNA target sequence that reduced transgene expression in DRG [5]. Moreover, a recent study comparing different , production methods contained an AAV9-only control that did not cause DRG toxicity, indicating that toxicity is mediated specifically by transgene overexpression [6]. Importantly, it may be challenging to directly compare the outcomes of studies in which similar, but slightly different, AAV types are used, as each AAV vector has its . . .
  

   

  

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