期刊
TRENDS IN MOLECULAR MEDICINE
卷 24, 期 8, 页码 669-681出版社
ELSEVIER SCI LTD
DOI: 10.1016/j.molmed.2018.06.006
关键词
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资金
- European Research Council (ERC) [694323]
- University of Naples Federico II under STAR
- European Research Council (ERC) [694323] Funding Source: European Research Council (ERC)
The retina has been at the forefront of translational gene therapy. Proof-ofconcept that gene therapy could restore vision in a large animal led to the initiation of the first successful clinical trials and, in turn, to the recent approval of the first gene therapy product for an ocular disease. As dozens of clinical trials of retinal gene therapy have begun, new challenges are identified, which include delivery of large genes, counteracting gain-offunction mutations, and safe and effective gene transfer to diseased retinas. Advancements in vector design, improvements of delivery routes, and selection of optimal timing for intervention will contribute to extend the initial success of retinal gene therapy to an increasing number of inherited blinding conditions.
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