Three-Year Follow-up after Unilateral Subretinal Delivery of Adeno-Associated Virus in Patients with Leber Congenital Amaurosis Type 2
| العنوان: | Three-Year Follow-up after Unilateral Subretinal Delivery of Adeno-Associated Virus in Patients with Leber Congenital Amaurosis Type 2 |
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| المؤلفون: | J. Fraser Wright, Alberto Auricchio, Katherine A. High, Paolo Melillo, Albert M. Maguire, Francesca Simonelli, Eric A. Pierce, Jennifer Wellman, Kathleen A. Marshall, Sandro Banfi, Francesco Testa, Settimio Rossi, Jean Bennett, Carmela Acerra, Junwei Sun, Enrico Maria Surace |
| المساهمون: | Testa, F, Maguire, Am, Rossi, S, Pierce, Ea, Melillo, P, Marshall, K, Banfi, S, Surace, Enrico Maria, Sun, J, Acerra, C, Wright, Jf, Wellman, J, High, Ka, Auricchio, Alberto, Bennett, J, Simonelli, F., Testa, Francesco, Rossi, Settimio, Melillo, Paolo, Banfi, Sandro, Surace, Em, Wright, Fj, Auricchio, A, Simonelli, Francesca |
| المصدر: | Ophthalmology. 120:1283-1291 |
| بيانات النشر: | Elsevier BV, 2013. |
| سنة النشر: | 2013 |
| مصطلحات موضوعية: | Adult, Male, cis-trans-Isomerases, medicine.medical_specialty, Visual acuity, Adolescent, genetic structures, Genetic Vectors, Leber Congenital Amaurosis, Visual Acuity, Nystagmus, Gene mutation, Reflex, Pupillary, Transfection, Article, Young Adult, Ophthalmology, medicine, Humans, Pupillary light reflex, Child, business.industry, Genetic Therapy, Dependovirus, eye diseases, Visual field, Clinical trial, Cis-trans-Isomerases, Mutation, Cohort, Female, Injections, Intraocular, Visual Fields, medicine.symptom, business, Tomography, Optical Coherence, Follow-Up Studies |
| الوصف: | OBJECTIVE: The aim of this study was to show the clinical data of long-term (3-year) follow-up of 5 patients affected by Leber congenital amaurosis type 2 (LCA2) treated with a single unilateral injection of adeno-associated virus AAV2-hRPE65v2. DESIGN: Clinical trial. PARTICIPANTS: Five LCA2 patients with RPE65 gene mutations. METHODS: After informed consent and confirmation of trial eligibility criteria, the eye with worse visual function was selected for subretinal delivery of adeno-associated virus (AAV2-hRPE65v2). Subjects were evaluated before and after surgery at designated follow-up visits (1, 2, 3, 14, 30, 60, 90, 180, 270, and 365 days, 1.5 years, and 3 years) by complete ophthalmic examination. Efficacy for each subject was monitored with best-corrected visual acuity, kinetic visual field, nystagmus testing, and pupillary light reflex. MAIN OUTCOME MEASURES: Best-corrected visual acuity, kinetic visual field, nystagmus testing, and pupillary light reflex. RESULTS: The data showed a statistically significant improvement of best-corrected visual acuity between baseline and 3 years after treatment in the treated eye (P |
| تدمد: | 0161-6420 |
| الوصول الحر: | https://explore.openaire.eu/search/publication?articleId=doi_dedup___::05a60be709fc62c35116da6cbfb1fb0dTest https://doi.org/10.1016/j.ophtha.2012.11.048Test |
| حقوق: | OPEN |
| رقم الانضمام: | edsair.doi.dedup.....05a60be709fc62c35116da6cbfb1fb0d |
| قاعدة البيانات: | OpenAIRE |
| تدمد: | 01616420 |
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