Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model
Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model
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DOI：
10.1126/science.1086137
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发表时间：
2003-08-08
期刊：
影响因子：
56.9
通讯作者：
Gage, FH
中科院分区：
文献类型：
--
作者：
Kaspar, BK;Lladó, J;Gage, FH
肌萎缩侧索硬化症（ALS）是一种进行性、致死性神经肌肉疾病，其与脊髓和脑干运动神经元的变性相关，导致肢体、轴肌和呼吸肌萎缩。ALS的病因尚不清楚，也没有有效的治疗方法。神经营养因子是ALS治疗评价的候选者。虽然慢性递送分子到中枢神经系统已被证明是困难的，但我们最近发现腺相关病毒可以有效地从肌肉逆行转运到脊髓的运动神经元。我们报告说，胰岛素样生长因子1延长寿命，延缓疾病进展，即使在明显的疾病症状时交付。
Amyotrophic lateral sclerosis (ALS) is a progressive, lethal neuromuscular disease that is associated with the degeneration of spinal and brainstem motor neurons, leading to atrophy of limb, axial, and respiratory muscles. The cause of ALS is unknown, and there is no effective therapy. Neurotrophic factors are candidates for therapeutic evaluation in ALS. Although chronic delivery of molecules to the central nervous system has proven difficult, we recently discovered that adeno-associated virus can be retrogradely transported efficiently from muscle to motor neurons of the spinal cord. We report that insulin-like growth factor 1 prolongs life and delays disease progression, even when delivered at the time of overt disease symptoms.