Gene therapy fully restores vision in mouse model of Leber congenital amaurosis
Monday, August 31, 2015 - 14:00
in Health & Medicine
Mice lacking the protein retGC1, which is deficient in humans suffering Leber congenital amaurosis-1 (LCA1), a disorder that causes severe visual impairment beginning in infancy, received gene therapy to replace retGC1 and showed fully restored visual function that persisted for at least 6 months. The success of this approach strongly support clinical testing of a gene therapy targeted to the retinas of LCA1 patients, conclude the authors of the study published in Human Gene Therapy. The article is available free on the Human Gene Therapy website until September 30, 2015.