Development of a transgenic animal model of spinocerebellar ataxia type 2
José A. Acosta, Ernesto L. Mola, Ricardo S. Rodríguez, Boris E. Acevedo, Luis H. Martínez.
Havana , February 3, 2006 . A new animal model has been generated at the Centre for Genetic Engineering and Biotec hnology (CIGB), Havana, Cuba, for studying spinocerebellar ataxia type 2 (SCA2) and evaluation of therapeutics for avoiding or delaying the onset of this incurable disease in humans. This transgenic mouse shares similar features to those of SCA2 patients (Neuroscience Letters, 2006, 392 : 202-206), and the CIGB is interested on testing New Molecular Entities in this animal model. The model is superior to the previously reported by Pulst et al. (Nature Genetics, 2000,26: 44-50) as symptoms appears early in the animals.
Animals can be used as models of human disease. Scientists can then use the animal model to study the way the disease progresses and what factors are important to the disease process. The model can also be used to study disease treatment, through the use of controlled experiments. Human clinical studies are invariably performed before a new treatment is available for use by the public, but the drug must be tested in animal models to obtain enough certainty on the safety and efficacy. |
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The study of the normal or sick human brains can provide important insights into the pathogenesis of the polyglutamine disorders, but such observations are limited to the use of post-mortem brains. The generation of mouse models for the neurodegenerative disorders can help to circumvent this problem, but many of them are based in the expression of truncated constructs to produce neurodegeneration. In addition, most polyglutamine disease models use heterologous promoters to direct the expression of the transgene to the target of these diseases. In a close collaboration, Cuban scientists from the CIGB, the CIRAH (Holguín), and the IFAL ( Havana ) have obtained a transgenic mouse model based on the expression of the full-length SCA2 cDNA with a CAG repeat expansion under the regulation of the self-human SCA2 promoter. This innovative result will allow faster advances in the field of Neurosciences.
About the CIGB
The CIGB, a vanguard institution in the Life Sciences in Cuba , is a large research-production complex, endowed with the state of the art equipment, production capacities and personnel devoted to the development of new biotechnology products in all stages, from the cloning and expression using recombinant DNA technology to industrial production. The CIGB has extensive experience in the recombinant DNA technology dated back to 1980, with more than 1300 employees, owning the most modern and efficient technology, carefully studied-beforehand facilities so as to ease the quiet and continuous workflow with a total area of more than 70 000 m 2 . The CIGB is looking for corporate partnership for joint development of R&D projects.
About the CIRAH
The Center for Investigation and Rehabilitation of Hereditary Ataxia (CIRAH), assumes all medical cares of hereditary ataxia patients and their descendants at risk.. This institution performs scientific research and develops specific programs of rehabilitation.
About the IFAL
Major activities at the Food and Pharmacy Institute (IFAL) are: i-) to graduate high-level pharmaceutical and food sciences professionals, ii-) to upgrade the knowledge of professionals and performing Master and Doctorate programs, and iii-) to develop research-development projects in collaboration with national scientific institutions.
For information: http://gndp.cigb.edu.cu or http://www.cigb.edu.cu
Contact: ernesto.lopez@cigb.edu.cu
Phone: (53-7) 271 2397. |
