Expression of truncated dystrophin cDNAs mediated by a lentiviral vector.
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.


OBJECTIVE
To explore the expression feasibility of truncated dystrophin cDNAs mediated by a lentiviral vector derived from feline immunodeficiency virus.


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 293Ad5+ 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.
Shunchang
Sun
S
Department of Medical Laboratory Science, Shenzhen Baoan Hospital, Southern Medical University, Shenzhen, Guangdong, China.
Haitao
Chen
C
Weidong
Chen
C
Jingbo
He
H
Yunsheng
Peng
P
D002461:Cell Line, Transformed; D016189:Dystrophin / Q000235:genetics* / Q000378:metabolism*; D015870:Gene Expression / Q000235:genetics*; D005822:Genetic Vectors / Q000502:physiology*; D006801:Humans; D016086:Lentivirus / Q000235:genetics*; D032446:Myoblasts / Q000378:metabolism; D011993:Recombinant Fusion Proteins / Q000235:genetics / Q000378:metabolism; D014162:Transfection / Q000379:methods
D016189:Dystrophin; D011993:Recombinant Fusion Proteins