title: Expression of truncated dystrophin cDNAs mediated by a lentiviral vector.
abstract: 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.
authors.affiliation: Department of Medical Laboratory Science, Shenzhen Baoan Hospital, Southern Medical University, Shenzhen, Guangdong, China.
authors.forename: Sun
authors.initials: S
authors.lastname: Shunchang
authors.forename: Chen
authors.initials: C
authors.lastname: Haitao
authors.forename: Chen
authors.initials: C
authors.lastname: Weidong
authors.forename: He
authors.initials: H
authors.lastname: Jingbo
authors.forename: Peng
authors.initials: P
authors.lastname: Yunsheng
mesh_terms: 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
chemical_list: D016189:Dystrophin; D011993:Recombinant Fusion Proteins