Key result
Adenoviral CT-1 gene therapy extends life-span by ~18% in pmn mice while reducing motor neuron degeneration.
Why the study?
Progressive motor neuronopathy (pmn) mice suffer from motoneuronal degeneration and premature death, and existing neurotrophic factors have not prevented disease onset or progression, motivating investigation of cardiotrophin-1 (CT-1) as a potential therapeutic agent.
Population
Newborn pmn mice with progressive motoneuronal degeneration
Comparison
Adenoviral CT-1 gene transfer vs untreated or AdLacZ-injected pmn mice
Design
Intramuscular adenoviral gene transfer study in neonatal mice, single-blind
Follow-up
Up to 35 days of age
Authors
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Supports further preclinical CT-1 gene therapy testing in motor neuron models; leaves open human translation from this animal study.
Effect estimate: 18% increase in mean life-span
Absolute Event Rate: 48.2% vs 40.8%
p-value: p=0.0012
Bordet et al. (1999) studied Progressive motor neuronopathy (n=38). Adenoviral cardiotrophin-1 (AdCT-1) gene transfer vs. AdLacZ-injected pmn mice was evaluated on Mean life-span (18% increase in mean life-span, p=0.0012). Intramuscular administration of an adenoviral CT-1 vector to newborn pmn mice increased mean life-span by 18% compared to control vector (48.2 vs 40.8 days, P=0.0012) and reduced motor neuron degeneration.
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