Intraparenchymal spinal cord delivery of adeno-associated virus IGF-1 is protective in the SOD1G93A model of ALS

Angelo C. Lepore, Christine Haenggeli, Mehdi Gasmi, Kathie M. Bishop, Raymond T. Bartus, Nicholas J. Maragakis, Jeffrey D. Rothstein

Research output: Contribution to journalArticlepeer-review


The potent neuroprotective activities of neurotrophic factors, including insulin-like growth factor 1 (IGF-1), make them promising candidates for treatment of amyotrophic lateral sclerosis (ALS). In an effort to maximize rate of motor neuron transduction, achieve high levels of spinal IGF-1 and thus enhance therapeutic benefit, we injected an adeno-associated virus 2 (AAV2)-based vector encoding human IGF-1 (CERE-130) into lumbar spinal cord parenchyma of SOD1G93A mice. We observed robust and long-term intraspinal IGF-1 expression and partial rescue of lumbar spinal cord motor neurons, as well as sex-specific delayed disease onset, weight loss, decline in hindlimb grip strength and increased animal survival.

Original languageEnglish (US)
Pages (from-to)256-265
Number of pages10
JournalBrain research
Issue number1
StatePublished - Dec 14 2007


  • Adeno-associated virus
  • Amyotrophic lateral sclerosis
  • Gene therapy
  • Insulin-like growth factor 1
  • Neurodegeneration
  • Neuroprotection

ASJC Scopus subject areas

  • Neuroscience(all)
  • Molecular Biology
  • Clinical Neurology
  • Developmental Biology


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