NDLI: Lentivector-mediated SMN replacement in a mouse model of spinal muscular atrophy

Content Provider	PubMed Central
Author	Azzouz, Mimoun Le, Thanh Ralph, G. Scott Walmsley, Lucy Monani, Umrao R. Lee, Debbie C. P. Wilkes, Fraser Mitrophanous, Kyriacos A. Kingsman, Susan M. Burghes, Arthur H. M. Mazarakis, Nicholas D.
Copyright Year	2004
Abstract	Spinal muscular atrophy (SMA) is a frequent recessive autosomal disorder. It is caused by mutations or deletion of the telomeric copy of the survival motor neuron (SMN) gene, leading to depletion in SMN protein levels. The treatment rationale for SMA is to halt or delay the degeneration of motor neurons, but to date there are no effective drug treatments for this disease. We have previously demonstrated that pseudotyping of the nonprimate equine infectious anemia virus (using the lentivector gene transfer system) with the glycoprotein of the Evelyn-Rokitnicki-Abelseth strain of the rabies virus confers retrograde axonal transport on these vectors. Here, we report that lentivector expressing human SMN was successfully used to restore SMN protein levels in SMA type 1 fibroblasts. Multiple single injections of a lentiviral vector expressing SMN in various muscles of SMA mice restored SMN to motor neurons, reduced motor neuron death, and increased the life expectancy by an average of 3 and 5 days (20% and 38%) compared with LacZ and untreated animals, respectively. Further extension of survival by SMN expression constructs will likely require a knowledge of when and/or where high levels of SMN are needed.
Related Links	http://dx.doi.org/10.1172/jci22922
Ending Page	1731
Page Count	6
Starting Page	1726
File Format	PDF
ISSN	00219738
Journal	Journal of Clinical Investigation
Issue Number	12
Volume Number	114
Language	English
Publisher	American Society for Clinical Investigation
Publisher Date	2004-12-15
Access Restriction	Open
Rights Holder	American Society for Clinical Investigation
Subject Keyword	Medicine(all) Research in Higher Education
Content Type	Text
Resource Type	Article
Subject	Medicine

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4	Project PI / Joint PI	Principal Investigator and Joint Principal Investigators of the project	Dr. B. Sutradhar bsutra@ndl.gov.in Prof. Saswat Chakrabarti will be added soon
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