Lundstrom Kenneth
Address Flamel Technologies, 33 Avenue du Dr Georges Lévy, 69693 Vénissieux, France.
Curr Opin Investig Drugs. 2007 Jan;8(1):34-40.
Neurological disorders represent a major challenge for therapeutic interventions due to the sensitivity and complexity of the CNS and the requirement of the therapeutic agent(s) to have a long-term effect. Gene therapy applications have opened up new possibilities to specifically deliver not only drugs but also small 'drug factories' by non-viral or viral vectors to the target site of action. A number of studies have demonstrated the feasibility of the gene therapy approach in animal models, and the simultaneous engineering of safer and more reliable vectors has moved the technology one step closer to clinical administration. In particular, viral vectors can provide long-term expression extended over one year. Most promisingly, the discovery of RNA interference has provided a novel method of specific gene silencing, which presents the opportunity to develop new therapies for the treatment of neurological disorders.
由于中枢神经系统的敏感性和复杂性，以及治疗药物需要具有长期效果，神经疾病对治疗干预构成了重大挑战。基因治疗应用开辟了新的可能性，不仅可以通过非病毒或病毒载体将药物，还可以将小型“药物工厂”特异性地递送至作用靶点。多项研究已证明基因治疗方法在动物模型中的可行性，同时开发更安全、更可靠的载体使该技术向临床应用又迈进了一步。特别是，病毒载体可以提供长达一年以上的长期表达。最有前景的是，RNA干扰的发现提供了一种特异性基因沉默的新方法，这为开发治疗神经疾病的新疗法提供了机会。
