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Nature:用基因疗法治疗先天视力缺陷

作者:生物谷来源:EurekAlert!2009-10-12 15:05:54分享到: 0
关键词: 视力缺陷 基因治疗

人们经常假设,视力和其他神经能力的发育存在关键时期,这些关键时期在青春期之前结束。例如,基因疗法对患有先天性视力障碍的***也许是不可能的。但用成年蜘蛛猿(它们正常情况下是红绿色盲)所做实验表明,有可能通过基因疗法向它们视网膜细胞中添加第三种光色素(人的视蛋白),从而使这些蜘蛛猿获得一个新维度的色视觉。这项工作不仅为人类的一种常见先天性视力缺陷提出了一个可能的疗法(临床试验目前正在进行当中),而且还表明视觉处理具有极大的神经可塑性,并为三色视觉的形成指出了可能的路径。(生物谷Bioon.com)

生物谷推荐原始出处:

Nature 461, 784-787 (8 October 2009) | doi:10.1038/nature08401

Gene therapy for red–green colour blindness in adult primates

Katherine Mancuso1, William W. Hauswirth2, Qiuhong Li2, Thomas B. Connor3, James A. Kuchenbecker1, Matthew C. Mauck3, Jay Neitz1 & Maureen Neitz1

1 Department of Ophthalmology, Box 356485, University of Washington, 1959 North East Pacific Street, Seattle, Washington 98195, USA
2 Department of Ophthalmology and Powell Gene Therapy Center, University of Florida, 1600 South West Archer Road, Gainesville, Florida 32610, USA
3 Department of Ophthalmology, Medical College of Wisconsin, 925 North 87th Street, Milwaukee, Wisconsin 53226, USA
4 Correspondence to: Jay Neitz1 Correspondence and requests for materials should be addressed to J.N.

Red–green colour blindness, which results from the absence of either the long- (L) or the middle- (M) wavelength-sensitive visual photopigments, is the most common single locus genetic disorder. Here we explore the possibility of curing colour blindness using gene therapy in experiments on adult monkeys that had been colour blind since birth. A third type of cone pigment was added to dichromatic retinas, providing the receptoral basis for trichromatic colour vision. This opened a new avenue to explore the requirements for establishing the neural circuits for a new dimension of colour sensation. Classic visual deprivation experiments1 have led to the expectation that neural connections established during development would not appropriately process an input that was not present from birth. Therefore, it was believed that the treatment of congenital vision disorders would be ineffective unless administered to the very young. However, here we show that the addition of a third opsin in adult red–green colour-deficient primates was sufficient to produce trichromatic colour vision behaviour. Thus, trichromacy can arise from a single addition of a third cone class and it does not require an early developmental process. This provides a positive outlook for the potential of gene therapy to cure adult vision disorders.

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