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Safe and effective superoxide dismutase 1 silencing using artificial microRNA in macaques.
Florie Borel1,2, Gwladys Gernoux1, Huaming Sun1
1Horae Gene Therapy Center, University of Massachusetts Medical School, 368 Plantation Street, Worcester, MA 01605, USA.
Science Translational Medicine
|November 2, 2018
Summary
Gene therapy using artificial microRNA targeting SOD1 shows promise for treating familial Amyotrophic Lateral Sclerosis (ALS). This approach effectively silenced the SOD1 gene in non-human primates, supporting its potential for clinical development.
Area of Science:
- Neuroscience
- Genetics
- Gene Therapy
Background:
- Amyotrophic lateral sclerosis (ALS) is a fatal motor neuron disease.
- Approximately 10% of ALS cases stem from dominant gain-of-function mutations in genes like SOD1.
- Gene silencing is a potential therapeutic strategy for mutant SOD1-linked ALS.
Purpose of the Study:
- To preclinically evaluate the safety and efficacy of adeno-associated virus (AAV)-mediated artificial microRNA (miRNA) gene silencing of SOD1 in a non-human primate model.
- To optimize AAV delivery to the spinal cord for treating mutant SOD1-related ALS.
Main Methods:
- Utilized an adeno-associated virus (AAV) encoding an artificial miRNA targeting SOD1.
- Administered AAV intrathecally to cynomolgus macaques, optimizing delivery via catheter and head-down positioning.
- Compared different promoters for artificial miRNA expression against mutant SOD1.
Main Results:
- Demonstrated efficient AAV delivery to the spinal cord in macaques.
- Achieved effective silencing of the SOD1 gene in motor neurons.
- Confirmed the safety of the AAV delivery method in non-human primates.
Conclusions:
- AAV-mediated artificial miRNA gene therapy targeting SOD1 is safe and effective in a preclinical non-human primate model.
- This approach warrants further development for treating patients with mutant SOD1-linked Amyotrophic Lateral Sclerosis (ALS).
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