Gene therapy for red-green colour blindness in adult primates.
Gene therapy for red-green colour blindness in adult primates.
复制标题
成人灵长类动物红绿色色盲的基因疗法。
作者：
红绿色盲是最常见的单基因座遗传病，其原因是缺乏长波长（L）或中波长（M）敏感的视觉色素。在这里，通过对自出生以来就患有色盲的成年猴子进行实验，探索了使用基因疗法治疗色盲的可能性。第三种类型的视锥色素被添加到二色视网膜中，为三色色觉提供了受体基础。这开辟了一条新的途径，探索建立新的颜色感觉维度的神经回路的要求。经典的视觉剥夺实验已经导致了这样的预期，即在发育过程中建立的神经连接不会适当地处理出生时不存在的输入。因此，人们认为，除非对非常年幼的儿童进行治疗，否则先天性视力障碍的治疗将是无效的。然而，在这里，在成年红绿色盲灵长类动物中添加第三种视蛋白足以产生三色色觉行为。因此，三色视觉可以从第三种视锥细胞的单一增加中产生，并且不需要早期的发育过程。这为基因治疗治疗成人视力障碍的潜力提供了积极的前景。
Red-green colour blindness, which results from the absence of either the long- (L) or middle- (M) wavelength-sensitive visual photopigments, is the most common single locus genetic disorder. Here, the possibility of curing colour blindness using gene therapy was explored 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 experiments 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 treatment of congenital vision disorders would be ineffective unless administered to the very young. Here, however, 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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影响因子：
2.5
作者：
WIESEL, TN;HUBEL, DH
通讯作者：
HUBEL, DH
DOI：
10.1073/pnas.0807027105
发表时间：
2008-09-30
影响因子：
11.1
作者：
Cideciyan, Artur V.;Aleman, Tomas S.;Hauswirth, William W.
通讯作者：
Hauswirth, William W.
影响因子：
1.8
作者：
Jacobs, GH
通讯作者：
Jacobs, GH
影响因子：
1.9
作者：
Kuchenbecker, James A.;Sahay, Manisha;Neitz, Jay
通讯作者：
Neitz, Jay
影响因子：
158.5
作者：
Maguire, Albert M.;Simonelli, Francesca;Bennett, Jean
通讯作者：
Bennett, Jean