Distinctive gene transduction efficiencies of commonly used viral vectors in the retina.
The transduction efficiency and cell tropism of viral vectors rAAV2/1, rAAV2, Ad5, Ad5/F35, and Lentivirus were evaluated in retina. All viral vectors achieved efficient transduction in living rat retina. However, each vector showed distinctive efficiency in vitro especially for rAAV2/1, which displayed poor transduction in cultured retinal cells. Distinctive cell-specific GFP expression was observed in vivo and in vitro for the same viral vector. The cell-specific tropism was not strictly correlated with the correspondent distribution of viral receptors in retina. These results provided important insights into the selection of appropriate vectors when specific retinal diseases are considered for gene therapy.
Zhang
Sheng-Hai
SH
Experimental Research Center, No.1 People's Hospital, Shanghai Jiaotong University, Shanghai, China.
Wu
Ji-Hong
JH
Wu
Xiao-Bing
XB
Dong
Xiao-Yan
XY
Liu
Xin-Jian
XJ
Li
Chuan-Yuan
CY
Qian-Huang
D000256:Adenoviridae / Q000235:genetics*; D000328:Adult; D000818:Animals; D002478:Cells, Cultured; D062727:Coxsackie and Adenovirus Receptor-Like Membrane Protein; D000229:Dependovirus / Q000235:genetics*; D015870:Gene Expression; D017930:Genes, Reporter; D005822:Genetic Vectors; D049452:Green Fluorescent Proteins / Q000235:genetics; D015497:HIV-1 / Q000235:genetics*; D019812:Heparan Sulfate Proteoglycans / Q000378:metabolism; D006801:Humans; D007124:Immunoenzyme Techniques; D016023:Integrins / Q000378:metabolism; D051916:Membrane Cofactor Protein / Q000378:metabolism; D010857:Pigment Epithelium of Eye / Q000378:metabolism; D051381:Rats; D017207:Rats, Sprague-Dawley; D011991:Receptors, Virus / Q000378:metabolism; D019030:Receptors, Vitronectin / Q000378:metabolism; D012160:Retina / Q000378:metabolism*; D014161:Transduction, Genetic; D018523:Tropism
C568771:CLMP protein, human; C568773:Clmp protein, rat; D062727:Coxsackie and Adenovirus Receptor-Like Membrane Protein; D019812:Heparan Sulfate Proteoglycans; D016023:Integrins; D051916:Membrane Cofactor Protein; D011991:Receptors, Virus; D019030:Receptors, Vitronectin; C064660:integrin alphaVbeta5; D049452:Green Fluorescent Proteins