title: [Gene expression of AAV-ITR ssDNA mini vector in skeletal muscle of mice].
abstract: AAV-ITR single strand DNA mini vector (AAV-ITR ssDNA mini vector) is a novel gene expression vector based on AAV-ITR. We have shown efficient gene expression of AAV-ITR ssDNA mini vector in HEK 293T. Here, we studied the efficacy of gene expression of AAV-ITR ssDNA mini vector in vivo. We injected the skeletal muscle of ICR mice separately with equal molars of AAV-ITR ssDNA mini vector, ITR mutated AAV-ITR single strand DNA mini vector (AAV-ITRmm ssDNA mutant vector), AAV-ITR dsDNA and pUC57-minivector-GFP, combined with TurboFect. Florescence microscope analysis of skeletal muscle section shows that AAV-ITR ssDNA mini vector had higher expression efficiency and longer expression period. We extracted DNA from the muscle three months after injection and quantified three vectors by Real-time PCR. RT-PCR analysis shows that there were highest copy numbers of AAV-ITR ssDNA mini vector existing in muscle. Stable existing of AAV- TR ssDNA mini vector in muscle could be the molecular basis of long term gene expression of the vector. The results suggest that AAV-ITR ssDNA mini vector might be a promising vector for gene therapy.
authors.forename: Dongqin
authors.initials: D
authors.lastname: Zhu
authors.forename: Yun
authors.initials: Y
authors.lastname: Zhang
authors.forename: Xiaomei
authors.initials: X
authors.lastname: Liu
authors.forename: Chun
authors.initials: C
authors.lastname: Zhang
mesh_terms: D000818:Animals; D004277:DNA, Single-Stranded / Q000235:genetics*; D000229:Dependovirus / Q000235:genetics*; D015870:Gene Expression; D015316:Genetic Therapy; D005822:Genetic Vectors; D051379:Mice; D008813:Mice, Inbred ICR; D018482:Muscle, Skeletal / Q000378:metabolism*; D060888:Real-Time Polymerase Chain Reaction
chemical_list: D004277:DNA, Single-Stranded