Improvement and Decline in Vision with Gene Therapy in Childhood Blindness
نویسندگان
چکیده
منابع مشابه
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چکیده ندارد.
Gene therapy for blindness.
Sight-restoring therapy for the visually impaired and blind is a major unmet medical need. Ocular gene therapy is a rational choice for restoring vision or preventing the loss of vision because most blinding diseases originate in cellular components of the eye, a compartment that is optimally suited for the delivery of genes, and many of these diseases have a genetic origin or genetic component...
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متن کاملIn utero gene therapy rescues vision in a murine model of congenital blindness.
The congenital retinal blindness known as Leber congenital amaurosis (LCA) can be caused by mutations in the RPE65 gene. RPE65 plays a critical role in the visual cycle that produces the photosensitive pigment rhodopsin. Recent evidence from human studies of LCA indicates that earlier rather than later intervention may be more likely to restore vision. We determined the impact of in utero deliv...
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ژورنال
عنوان ژورنال: New England Journal of Medicine
سال: 2015
ISSN: 0028-4793,1533-4406
DOI: 10.1056/nejmoa1412965