Adeno-associated virus (AAV) based gene therapy for eye diseases
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Adeno-associated virus (AAV) based gene therapy for eye diseases Shuang Wang • Peng Liu • Lei Song • Lei Lu Wensong Zhang • Yazhen Wu
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Received: 12 December 2010 / Accepted: 4 February 2011 / Published online: 23 February 2011 Ó Springer Science+Business Media B.V. 2011
Abstract Gene therapy emerged as important approach in treatment for many inborn disorders caused by genetic defects, as well as other diseases. This manuscript focused on Adeno-associated virus (AAV) based gene therapy to eye diseases. The paper firstly introduced the AAV vectors and the techniques of eye delivery, then summarized some tested genes that were used in past treatment to retinal degeneration disorders. Finally the paper discussed the updated optogenetics and its roles in AAV based gene therapy for eye diseases. Keywords AAV Gene therapy Optogenetics Neovascularization Trophic factor Neuroprotection
Introduction The Adeno-associated virus (AAV) is a small virus that can infect both humans and some other primate S. Wang Y. Wu (&) Department of Ophthalmology, The Second Hospital of Ji Lin University, 130041 Ji Lin, China e-mail: [email protected] S. Wang P. Liu China-Japan Union Hospital of Jilin University, Ji Lin, China L. Song L. Lu W. Zhang Department of Neurology, The First Hospital of Ji Lin University, Ji Lin, China
species. The facts that AAV does not contribute to any known disease, and it only raises mild immune response following host infection suggest AAV as very attractive vector candidate for gene therapy in clinical applications. Additionally, AAV could infect both dividing and non-dividing cells with the potential of genome integration; however, it lacks the threat of random insertion into the genome as observed in retrovirus, which could cause cancer in sometimes. In gene therapy application, the AAV vector was modified to eliminate the integrative capacity by removing specific sequences from the DNA. This made AAV as one of the most popular genetic vectors being used. Gene therapy is not new. The idea emerged shortly after the understanding of the structure of DNA and became into reality with trails in transfecting human cell lines with external DNA constructs in 1970s (Friedmann and Roblin 1972). Then the first approved gene therapy case in the United States was in 1990, performed on a girl with genetic defects leading to immune system deficiency. The efforts of gene therapy to treat eye diseases also started in 1990s (da Cruz et al. 1997; Murata et al. 1997; Wright 1997). Several possibilities have been proposed to treat age-resulted eye degeneration or blindness due to genetic defects: the first is the direct injection of viral genetic vectors expressing therapeutic genes; second is the injection of non-viral vectors, which could be delivered into the soma with physical methods, as in electroporation; third is the
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Cell Tissue Bank (2011) 12:105–110
transplantation of genetically-modified cells that would replace the abnormal tissues. In present manuscript we would like to focus on th
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