Effect of Gene Therapy on Visual Function in Leber's Congenital Amaurosis
نویسندگان
چکیده
منابع مشابه
Effect of gene therapy on visual function in Leber's congenital amaurosis.
Early-onset, severe retinal dystrophy caused by mutations in the gene encoding retinal pigment epithelium-specific 65-kD protein (RPE65) is associated with poor vision at birth and complete loss of vision in early adulthood. We administered to three young adult patients subretinal injections of recombinant adeno-associated virus vector 2/2 expressing RPE65 complementary DNA (cDNA) under the con...
متن کاملHuman retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement.
Leber congenital amaurosis (LCA) associated with retinal pigment epithelium-specific protein 65 kDa (RPE65) mutations is a severe hereditary blindness resulting from both dysfunction and degeneration of photoreceptors. Clinical trials with gene augmentation therapy have shown partial reversal of the dysfunction, but the effects on the degeneration are not known. We evaluated the consequences of...
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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 کاملLebers Amaurosis in Three Siblings: A case report
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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abstract the present study tried to investigate the effect of visual aids (films) on the development of the speech act of disagreement among iranian efl intermediate learners. to this end, the researcher selected 40 homogeneous intermediate learners based on their scores on oxford placement test. .the subjects then divided into control group and experimental group. both classes were tested by ...
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ژورنال
عنوان ژورنال: New England Journal of Medicine
سال: 2008
ISSN: 0028-4793,1533-4406
DOI: 10.1056/nejmoa0802268