Nonviral vectors offer a safer alternative to viral vectors for neuronal gene delivery, but their clinical utility remains limited by low transfection efficiencies and multiple biological barriers.
This review highlights the challenges and strategies for improving nonviral delivery of nucleic acids to neurons for treating neurological diseases.
The delivery of therapeutic nucleic acids to neurons has the potential to treat neurological disease and spinal cord injury. While select viral vectors have shown promise as gene carriers to neurons, their potential as therapeutic agents is limited by their toxicity and immunogenicity, their broad tropism, and the cost of large-scale formulation. Nonviral vectors are an attractive alternative in that they offer improved safety profiles compared to viruses, are less expensive to produce, and can be targeted to specific neuronal subpopulations. However, most nonviral vectors suffer from significantly lower transfection efficiencies than neurotropic viruses, severely limiting their utility in neuron-targeted delivery applications. To realize the potential of nonviral delivery technology in neurons, vectors must be designed to overcome a series of extra- and intracellular barriers. In this article, we describe the challenges preventing successful nonviral delivery of nucleic acids to neurons and review strategies aimed at overcoming these challenges.
Bergen et al. (Thu,) conducted a review in Neurological disease and spinal cord injury. Nonviral vectors for nucleic acid delivery vs. Viral vectors was evaluated. Nonviral vectors offer a safer alternative to viral vectors for neuronal gene delivery, but their clinical utility remains limited by low transfection efficiencies and multiple biological barriers.