Mendell Jerry R, Clark K Reed
Center for Gene Therapy, Columbus Children's Research Institute, Columbus, OH 43205, USA.
Curr Neurol Neurosci Rep. 2006 Jan;6(1):47-56. doi: 10.1007/s11910-996-0009-8.
Gene therapy for muscular dystrophy represents a promising avenue of pursuit for a disease with a limited repertoire of treatment. Recent successes in the research arena using adeno-associated viral vectors should accelerate the movement of gene-based therapeutics for muscle disorders into the clinic. Nevertheless, significant challenges remain before gene therapy can deliver on the promises avowed by early pioneers of the field. This review examines recent progress and the hurdles remaining to achieve gene-based treatment therapies for muscular dystrophy.
对于治疗方法有限的肌肉萎缩症而言，基因治疗是一条很有前景的探索途径。近期在研究领域使用腺相关病毒载体所取得的成功，应能加速基于基因的肌肉疾病治疗方法进入临床应用。然而，在基因治疗能够兑现该领域早期先驱者所宣称的承诺之前，仍存在重大挑战。这篇综述探讨了在实现基于基因的肌肉萎缩症治疗方法方面的最新进展以及尚存的障碍。
