Retinal gene delivery by rAAV and DNA electroporation.
Retinal gene delivery by rAAV and DNA electroporation.
复制标题
DOI：
10.1002/9780471729259.mc14d04s28
复制
发表时间：
2013
影响因子：
--
通讯作者：
Punzo, Claudio
中科院分区：
文献类型：
--
作者：
Venkatesh, Aditya;Ma, Shan;Langellotto, Fernanda;Gao, Guangping;Punzo, Claudio
Ocular gene therapy is a fast growing area of research. The eye is an ideal organ for gene therapy since it is immune privileged, easily accessible, and direct viral delivery results primarily in local infection. Because the eye is not a vital organ, mutations in eye specific genes tend to be more common. To date, over 40 eye specific genes have been identified which harbor mutations that lead to blindness. Gene therapy with recombinant Adeno Associated Virus (rAAV) holds the promise to treat patients with such mutations. However, proof-of-concept and safety evaluation for gene therapy remains to be established for most of these diseases. This unit describes the in vivo delivery of genes to the mouse eye by rAAV-mediated gene transfer and plasmid DNA electroporation. Advantages and limitations of these methods are discussed, and detailed protocols for gene delivery, required materials, as well as subsequent tissue processing methods are described.
