干细胞研究与治疗杂志

抽象的

Alphavirus Vectors for Therapy of Neurological Disorders

Kenneth Lundstrom

Alphavirus vectors engineered for gene delivery and expression of heterologous proteins have been considered as valuable tools for research on neurological disorders. They possess a highly efficient susceptibility for neuronal cells and can provide extreme levels of heterologous gene expression. However, they generally generate short-term transient expression, which might limit their therapeutic use in many neurological disorders often requiring long-term even life-long presence of therapeutic agents. Recent development in gene silencing applying both RNA interference and microRNA approaches will certainly expand the application range. Moreover, alphaviruses provide interesting models for neurological diseases such as demyelinating and spinal motor diseases.