Systemic Delivery of scAAV9 Expressing SMN Prolongs Survival in a Model of Spinal Muscular Atrophy
Abstract
An adeno-associated virus vector expressing the survival motor neuron protein rescues mice with spinal muscular atrophy.
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The ability of the recombinant AAV genome to persist as an episome in post-mitotic cells, such as neurons, ensures long-term expression even after a single administration.
“Especially considering the ability of the recombinant AAV genome to persist as an episome in post-mitotic cells, such as neurons, which ensures long-term expression even after a single administration [130-132].”
From Circumventing the packaging limit of AAV-mediated gene replacement therapy for neurological disorders (2022) · cites this paper as [131]
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References 29 · Cited by 285
- Authors
- Valori, C. F., Ning, K., Wyles, M., Mead, R. J., Grierson, A. J., Shaw, P. J., & Azzouz, M.
- Affiliations
- University of Sheffield (GB)
- Corresponding author
- Valori, Chiara F.
- Ning, Ke
- Azzouz, Mimoun
- Venue
- Science Translational Medicine
- Published
- 2010-06-09
- Volume
- 2
- Issue
- 35
- Pages
- 35ra42-35ra42
- ISSN
- 1946-6234 (ISSN-L)
- 1946-6242
- OpenAlex
- W2147552101
- Cited by
- 285
- References
- 29
- Citations by year
- 2026: 5 · 2025: 1 · 2024: 19 · 2023: 13 · 2022: 11 · 2021: 15 · 2020: 18 · 2019: 20 · 2018: 11 · 2017: 23 · 2016: 18 · 2015: 25 · 2014: 26 · 2013: 26 · 2012: 15
- FWCI
- 11.43
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Science Translational Medicine (publishedVersion)
- PubMed (publishedVersion)
- Topics
- Health Sciences › Medicine › Genetics › 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, SMN1, gene therapy, systemic gene delivery, survival motor neuron protein, transgenic mouse model, lifespan extension, motor neurons
- MeSH terms
- Dependovirus, Animals, Animals, Newborn, Base Sequence, Codon, Disease Models, Animal, Fibroblasts, Molecular Sequence Data, Motor Activity, Muscular Atrophy, Spinal, Mutation, Phenotype, Genetic Therapy, Survival Analysis, Gene Transfer Techniques, Mice, Survival of Motor Neuron 1 Protein
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