Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis
نویسندگان
چکیده
منابع مشابه
Safety and efficacy of gene transfer for Leber's congenital amaurosis.
Leber's congenital amaurosis (LCA) is a group of inherited blinding diseases with onset during childhood. One form of the disease, LCA2, is caused by mutations in the retinal pigment epithelium-specific 65-kDa protein gene (RPE65). We investigated the safety of subretinal delivery of a recombinant adeno-associated virus (AAV) carrying RPE65 complementary DNA (cDNA) (ClinicalTrials.gov number, N...
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Retinitis pigmentosa (RP) is the generic name for a group of hereditary disorders characterized by progressive loss of photoreceptors and RPE function. The classical triad of retinitis pigmentosa is arteriolar attenuation, retinal bone spicule pigmentation and waxy disc pallor. Lebers congenital amaurosis (LCA) is the hereditary form of retinitis pigmentosa. It presents with blindness either at...
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We evaluated the safety and efficacy of an optimized adeno-associated virus (AAV; AAV2.RPE65) in animal models of the RPE65 form of Leber congenital amaurosis (LCA). Protein expression was optimized by addition of a modified Kozak sequence at the translational start site of hRPE65. Modifications in AAV production and delivery included use of a long stuffer sequence to prevent reverse packaging ...
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To the Editor: The discussion of donor assessment by Kotton et al. suggests that exclusion criteria from the Centers for Disease Control and Prevention (CDC) (Table 3 of the article) are used to reduce the likelihood of transmission of the human immunodeficiency virus (HIV). In fact, the CDC’s criteria do not have an exclusionary function but define a group of donors considered to have a high r...
متن کاملLeber's congenital amaurosis.
rig. renpheralfundus cS. We have had occasion to see a similar case, first in 1974 when the patient, a black female with no apparent vision but otherwise well, was 6 months old. At that time dilated fundus examination under anaesthesia (EUA) was normal. Repeat EUA in 1977 at age 4 years revealed bilateral peripheral fundus lesions essentially identical to those described by Chew et al. (Figs. 1...
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ژورنال
عنوان ژورنال: New England Journal of Medicine
سال: 2008
ISSN: 0028-4793,1533-4406
DOI: 10.1056/nejmoa0802315