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・ Retina horizontal cell
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Retinal gene therapy using lentiviral vectors
・ Retinal haemorrhage
・ Retinal homeobox protein Rx
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Retinal gene therapy using lentiviral vectors : ウィキペディア英語版
Retinal gene therapy using lentiviral vectors

Gene therapy using lentiviral vectors is being explored in early stage trials.
== Trials ==
In a Phase I clinical trial of three patients, two showed no improvement and one of them had some improvements. The study concluded further investigation is warranted for the use of the procedure to treat Leber's congenital amaurosis. Other early trials have been used to explore the treatments potential, including for therapeutic use of recombinant adeno-associated virus (rAAV) vectors. Many other possible viral vectors remain options for the treatment of various genetic disorders in the retina that lead to blindness. Retinal gene therapy using lentivirus vectors may be a way to treat a wider range of genetic disorders in the retina because of the various properties of the lentivirus that make it an attractive alternative to rAAV vectors.
Like rAAV vectors, lentiviral vectors offer many features that make it an excellent tool for molecular biology and possible medical treatments. Like many other vectors commonly used in the laboratory, lentiviral vectors allow for efficient transfer of foreign DNA (transgene) to target cells, long-lasting and stable expression of the foreign DNA, and a generally reduced ability to produce an immune response. Like many other retroviral vectors, lentiviral vectors do not possess any of their original DNA content, allowing as little provocation of the immune response as possible. Unlike many retroviral vectors, though, lentiviral vectors offer the advantage of being able to successfully introduce a transgene to target cells whether or not the target cells proliferate (many retroviral vectors require replicating DNA to insert themselves into the host genome).〔
An important consideration for the application of the lentiviral vector is the parent virus that gave rise to the vector. Not all lentiviral vectors are perfectly suited to every application, and sometimes it becomes necessary for the researcher to try work with a different lentiviral vector if one does not offer the desired transgene expression. Other times, it may be necessary to use another viral vector altogether. There are options to choose from between lentiviral vectors, though, and many popular lentiviral vectors have either a human immunodeficiency virus 1 (HIV-1) or equine infectious anemia virus (EIAV).

抄文引用元・出典: フリー百科事典『 ウィキペディア(Wikipedia)
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