Gene therapy for red-green colour blindness in adult primates

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 mon...

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Bibliographic Details
Published in:	Nature (London) 2009-10, Vol.461 (7265), p.784-787
Main Authors:	Mancuso, Katherine, Neitz, Jay, Neitz, Maureen, Hauswirth, William W, Connor, Thomas B, Li, Qiuhong, Kuchenbecker, James A, Mauck, Matthew C
Format:	Article
Language:	eng
Subjects:	Aging Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy Animals Applied cell therapy and gene therapy Biological and medical sciences Care and treatment Color blindness Color Perception - genetics Color Perception - physiology Color Vision - genetics Color Vision - physiology Color Vision Defects - congenital Color Vision Defects - genetics Color Vision Defects - physiopathology Color Vision Defects - therapy Female Females Gene therapy Genetic aspects Genetic Therapy Genetic Vectors - genetics Humans Male Medical research Medical sciences Monkeys & apes Monoclonal antibodies Ophthalmology Opsins - genetics Opsins - metabolism Primates Retina - cytology Retina - metabolism Saimiri - genetics Saimiri - physiology Transfusions. Complications. Transfusion reactions. Cell and gene therapy Transgenes - genetics Treatment Outcome Vision disorders
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Summary:	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 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 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.
ISSN:	0028-0836 1476-4687