Why the study?
Does intramuscular injection of a small interfering RNA-encoding virus preserve grip strength in mice with superoxide dismutase mutations?
Population
Mice expressing mutations in superoxide dismutase (amyotrophic lateral sclerosis model)
Design
Preclinical
Authors
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Does not support clinical use; extends proof-of-principle for AAV-siRNA gene therapy in ALS models.
Does intramuscular injection of a small interfering RNA-encoding virus preserve grip strength in mice with superoxide dismutase mutations?
Intramuscular injection of an AAV carrying siRNA targeting superoxide dismutase preserves grip strength in an ALS mouse model, providing proof of principle for this gene therapy approach.
Miller et al. (2005) studied this question.
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