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Neurology India
Medknow Publications on behalf of the Neurological Society of India
ISSN: 0028-3886
EISSN: 0028-3886
Vol. 56, No. 1, 2008, pp. 52-56
Bioline Code: ni08011
Full paper language: English
Document type: Research Article
Document available free of charge

Neurology India, Vol. 56, No. 1, 2008, pp. 52-56

 en Expression of truncated dystrophin cDNAs mediated by a lentiviral vector
Shunchang Sun; Haitao Chen; Weidong Chen; Jingbo He & Yunsheng Peng


Background: The success of Duchenne muscular dystrophy gene therapy requires promising tools for gene delivery and mini-gene cassettes that can express therapeutic levels of a functional protein.
Aims: To explore the expression feasibility of truncated dystrophin cDNAs mediated by a lentiviral vector derived from feline immunodeficiency virus.
Materials and Methods: Three truncated dystrophin cDNAs were constructed by PCR cloning, then these cDNAs were inserted into lentiviral vectors. Recombinant lentiviruses were generated by transient transfection of lentiviral vector constructs into 293Ad 5+ cells. Cultured myoblasts were then infected with recombinant lentiviruses. Expression of truncated dystrophin cDNAs was detected by Western blot analysis.
Results: Mediated by lentiviral vectors, three cDNAs constructed by PCR cloning expressed relative truncated dystrophins in cultured myoblasts.
Conclusions: Truncated dystrophin cDNAs can express themselves successfully mediated by feline immunodeficiency virus vectors. It offers the possibility of an approach utilizing truncated dystrophin cDNAs and lentiviral vectors toward gene therapy of Duchenne muscular dystrophy.

Duchenne muscular dystrophy, dystrophin, gene transfer, lentivirus, vector

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