Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model

Brian K. Kaspar, Jerònia Lladó, Nushin Sherkat, Jeffrey D. Rothstein, Fred H. Gage

Research output: Contribution to journalArticlepeer-review

715 Scopus citations


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.

Original languageEnglish (US)
Pages (from-to)839-842
Number of pages4
Issue number5634
StatePublished - Aug 8 2003

ASJC Scopus subject areas

  • General


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