Retrograde Transport and ALS Therapy

Science's STKE  12 Aug 2003:
Vol. 2003, Issue 195, pp. tw320-TW320
DOI: 10.1126/stke.2003.195.tw320

Amyotrophic lateral sclerosis (ALS) is a fatal disease characterized by degeneration of the motor neurons for which there is no effective therapy. Kaspar et al. now show in a mouse model of ALS that injecting an adeno-associated viral vector carrying the neurotrophic factor, insulin growth factor 1 (IGF-1), into mouse limb muscles delays disease progression and prolongs survival of the animals. Travel of the viral vector by retrograde transport along the motor neurons that innervate the muscles delivered IGF-1 to the neuronal cell bodies.

B. K. Kaspar, J. Lladó, N. Sherkat, J. D. Rothstein, F. H. Gage, Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model. Science 301, 839-842 (2003). [Abstract] [Full Text]