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See Latest ArticlesWhich Leber congenital amaurosis patients are eligible for gene therapy trials
Journal of American Association for Pediatric Ophthalmology and Strabismus , 10/23/09
Drack AV et al. – To be eligible for participation in current RPE65 gene therapy trials, patients' DNA must contain 2 correctly segregating alleles with an EPP = 2 or 3. Interpretation of DNA variants is complex; genetic misdiagnosis may lead to ineffective treatment in some patients and lack of treatment in others.
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Irish Journal of Medical Science, 12/18/09
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