Article ID | Journal | Published Year | Pages | File Type |
---|---|---|---|---|
1983536 | The International Journal of Biochemistry & Cell Biology | 2014 | 5 Pages |
Abstract
Recombinant virus mediated gene therapy of Leber's Congenital Amaurosis has provided a wide range of data on the utility of gene replacement therapy for recessive diseases. Studies to date demonstrate that gene therapy in the eye is safe and can result in long-term recovery of visual function, but they also highlight that further research is required to identify optimum intervention time-points, target populations and the compatibility of associate therapies. This article is part of a directed issue entitled: Regenerative Medicine: the challenge of translation.
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Authors
Elizabeth P. Rakoczy, Kristina Narfström,