The DOI matches its Crossref record; 48 of the 48 references it cites were found in a registry or an index.
Direct Muscle Delivery of GDNF With Human Mesenchymal Stem Cells Improves Motor Neuron Survival and Function in a Rat Model of Familial ALS
Open accessAbstract
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease in which there is a progressive loss of motor neurons and their connections to muscle, leading to paralysis. In order to maintain muscle connections in a rat model of familial ALS (FALS), we performed intramuscular transplantation with human mesenchymal stem cells (hMSCs) used as "Trojan horses" to deliver growth factors to the terminals of motor neurons and to the skeletal muscles. hMSCs engineered to secrete glial cell line-derived neurotrophic factor (hMSC-GDNF) were transplanted bilaterally into three muscle groups. The cells survived within the muscle, released GDNF, and significantly increased the number of neuromuscular connections and motor neuron cell bodies in the spinal cord at mid-stages of the disease. Further, intramuscular transplantation with hMSC-GDNF was found to ameliorate motor neuron loss within the spinal cord where it connects with the limb muscles receiving transplants. While disease onset was similar in all the animals, hMSC-GDNF significantly delayed disease progression, increasing overall lifespan by up to 28 days, which is one of the largest effects on survival noted for this rat model of FALS. This preclinical data provides a novel and practical approach toward ex vivo gene therapy for ALS.
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Cited by
Rising Stars: Astrocytes as a Therapeutic Target for ALS Disease
Izrael, M., Slutsky, S. G., & Revel, M. (2020) · Frontiers in Neuroscience · doi:10.3389/fnins.2020.00824 · Cited by 73
Mohan, R., Tosolini, A. P., & Morris, R. (2014) · Neuroscience · doi:10.1016/j.neuroscience.2014.05.045 · Cited by 48
Metadata
- Authors
- Suzuki, M., McHugh, J., Tork, C., Shelley, B., Hayes, A., Bellantuono, I., Aebischer, P., & Svendsen, C. N.
- Venue
- Molecular Therapy
- Publisher
- Elsevier BV
- Published
- 2008-12
- Volume
- 16
- Issue
- 12
- Pages
- 2002-2010
- ISSN
- 1525-0016 (ISSN-L)
- OpenAlex
- W2070542281
- Cited by
- 262
- References
- 48
- FWCI
- 9.04
- Type
- journal article
- Language
- en
- Open access
- hybrid
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- CoChat. (2026). Citation check: Direct Muscle Delivery of GDNF With Human Mesenchymal Stem Cells Improves Motor Neuron Survival and Function in a Rat Model of Familial ALS. https://app.cochat.ai/papers/10.1038/mt.2008.197
- The paper
- Suzuki, M., McHugh, J., Tork, C., Shelley, B., Hayes, A., Bellantuono, I., Aebischer, P., & Svendsen, C. N. (2008). Direct Muscle Delivery of GDNF With Human Mesenchymal Stem Cells Improves Motor Neuron Survival and Function in a Rat Model of Familial ALS. Molecular Therapy. https://doi.org/10.1038/mt.2008.197
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Bibliographic data: Semantic Scholar, OpenAlex, Crossref.
Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).
Semantic Scholar’s data is described in: Kinney, R., et al. (2023). The Semantic Scholar Open Data Platform. arXiv:2301.10140.
