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RETRACTED ARTICLE: Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN
Open accessAbstract
Spinal muscular atrophy (SMA), the most common autosomal recessive neurodegenerative disease affecting children, results in impaired motor neuron function. Despite knowledge of the pathogenic role of decreased survival motor neuron (SMN) protein levels, efforts to increase SMN have not resulted in a treatment for patients. We recently demonstrated that self-complementary adeno-associated virus 9 (scAAV9) can infect approximately 60% of motor neurons when injected intravenously into neonatal mice. Here we use scAAV9-mediated postnatal day 1 vascular gene delivery to replace SMN in SMA pups and rescue motor function, neuromuscular physiology and life span. Treatment on postnatal day 5 results in partial correction, whereas postnatal day 10 treatment has little effect, suggesting a developmental period in which scAAV9 therapy has maximal benefit. Notably, we also show extensive scAAV9-mediated motor neuron transduction after injection into a newborn cynomolgus macaque. This demonstration that scAAV9 traverses the blood-brain barrier in a nonhuman primate emphasizes the clinical potential of scAAV9 gene therapy for SMA.
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“Early treatment at postnatal day 2 extended the lifespan from 15 days to >250 days.11”
From Current clinical applications of in vivo gene therapy with AAVs (2021) · cites this paper as [11]
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“Preclinical studies, delivering scAAV9-SMN to SMA pups, demonstrated positive effects on survival, growth, and neuromuscular transmission.11,12”
From Current clinical applications of in vivo gene therapy with AAVs (2021) · cites this paper as [11]
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Adeno-associated virus vector as a platform for gene therapy delivery
Wang, D., Tai, P. W. L., & Gao, G. (2019) · Nature Reviews Drug Discovery · doi:10.1038/s41573-019-0012-9 · Cited by 2,444
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
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Current clinical applications of in vivo gene therapy with AAVs
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Metadata
- Authors
- Foust, K. D., Wang, X., McGovern, V. L., Braun, L., Bevan, A. K., Haidet, A. M., Le, T. T., Morales, P. R., Rich, M. M., Burghes, A. H. M., & Kaspar, B. K.
- Affiliations
- Nationwide Children's Hospital (US)
- Wright State University (US)
- The Ohio State University (US)
- Corresponding author
- Kaspar, Brian K
- ORCID
- Venue
- Nature Biotechnology
- Published
- 2010-02-28
- Volume
- 28
- Issue
- 3
- Pages
- 271-274
- ISSN
- 0733-222X (ISSN-L)
- 1087-0156
- 1546-1696
- PMC
- PMC2889698
- OpenAlex
- W2045117117
- Cited by
- 688
- References
- 30
- Citations by year
- 2026: 5 · 2025: 10 · 2024: 17 · 2023: 19 · 2022: 42 · 2021: 49 · 2020: 61 · 2019: 51 · 2018: 32 · 2017: 56 · 2016: 48 · 2015: 51 · 2014: 57 · 2013: 54 · 2012: 48
- FWCI
- 27.28
- Type
- journal article
- Language
- en
- Status
- Retracted
- Update notices
- retraction (2022-10-06)
- Open access
- green
- www.ncbi.nlm.nih.gov
- Locations
- Nature Biotechnology (publishedVersion)
- PubMed (publishedVersion)
- PubMed Central (acceptedVersion)
- CORE Scholar (Wright State University) (submittedVersion)
- Journal of Bioresource Management (submittedVersion)
- Funders
- National Institute of Neurological Disorders and Stroke (R21NS064328, RC2NS069476, P30NS045758, P01NS057228, R01NS038650, F31NS058224)
- Topics
- Health Sciences › Medicine › Genetics (clinical) › Neurogenetic and Muscular Disorders Research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Keywords
- spinal muscular atrophy, gene therapy, motor neurons, blood-brain barrier, cynomolgus macaques, mouse model, retraction
- MeSH terms
- Dependovirus, Animals, Animals, Newborn, Disease Models, Animal, Macaca fascicularis, Male, Mice, Transgenic, Microscopy, Fluorescence, Motor Neurons, Muscular Atrophy, Spinal, Phenotype, Gene Transfer Techniques, Green Fluorescent Proteins, Mice, Kaplan-Meier Estimate, Survival of Motor Neuron 1 Protein
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