Vezzani Annamaria
Mario Negri Institute for Pharmacological Research, Milan, Italy.
Epilepsy Curr. 2004 May-Jun;4(3):87-90. doi: 10.1111/j.1535-7597.2004.43001.x.
The generation of viral vectors, such as adeno-associated virus (AAV) and lentivirus, which are capable of stable transduction of neurons, offers an attractive strategy for introducing novel genes into the brain, resulting in a long-lasting production of specific proteins. An alternative approach to achieving transgene expression in brain is to graft cells that are genetically engineered to produce neuroactive substances. Neuroactive peptides, adenosine, and gamma-aminobutyric acid, are agents that can be delivered by gene and cell therapy with potential utility in epilepsy therapy.
能够稳定转导神经元的病毒载体，如腺相关病毒（AAV）和慢病毒的产生，为将新基因引入大脑提供了一种有吸引力的策略，从而能长期产生特定蛋白质。在大脑中实现转基因表达的另一种方法是移植经过基因工程改造以产生神经活性物质的细胞。神经活性肽、腺苷和γ-氨基丁酸是可以通过基因和细胞疗法递送的物质，在癫痫治疗中具有潜在用途。
