Gene therapy to rescue retinal degeneration caused by mutations in rhodopsin.
Gene therapy to rescue retinal degeneration caused by mutations in rhodopsin.
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DOI：
10.1007/978-1-4939-2330-4_25
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发表时间：
2015
期刊：
影响因子：
--
通讯作者：
Lewin AS
中科院分区：
文献类型：
--
作者：
Rossmiller BP;Ryals RC;Lewin AS
Retinal gene therapy has proven safe and at least partially successful in clinical trials and in numerous animal models. Gene therapy requires characterization of the progression of the disease and understanding of its genetic cause. Testing gene therapies usually requires an animal model that recapitulates the key features of the human disease, though photoreceptors and cells of the retinal pigment epithelium produced from patient-derived stem cells may provide an alternative test system for retinal gene therapy. Gene therapy also requires a delivery system that introduces the therapeutic gene to the correct cell type and does not cause unintended damage to the tissue. Current systems being tested in the eye are nanoparticles, pseudotyped lentiviruses, and adeno-associated virus (AAV) of various serotypes. Here, we describe the techniques of AAV vector design as well as the in vivo and ex vivo tests necessary for assessing the efficacy of retinal gene therapy to treat retinal degeneration caused by mutations in the rhodopsin gene.
