Gene therapy for red-green colour blindness in adult primates. Nature 2009 Oct 08;461(7265):784-7
Date
09/18/2009Pubmed ID
19759534Pubmed Central ID
PMC2782927DOI
10.1038/nature08401Scopus ID
2-s2.0-70349971731 (requires institutional sign-in at Scopus site) 255 CitationsAbstract
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.
Author List
Mancuso K, Hauswirth WW, Li Q, Connor TB, Kuchenbecker JA, Mauck MC, Neitz J, Neitz MAuthor
Thomas B. Connor MD Professor in the Ophthalmology and Visual Sciences department at Medical College of WisconsinMESH terms used to index this publication - Major topics in bold
AgingAnimals
Color Perception
Color Vision
Color Vision Defects
Female
Genetic Therapy
Genetic Vectors
Humans
Male
Opsins
Retina
Saimiri
Transgenes
Treatment Outcome
