Effect of gene therapy on visual function in Leber’s congenital amaurosis

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29. Differential Macular Morphology in Patients with RPE65 -, CEP290 -, GUCY2D -, and AIPL1 -Related Leber Congenital Amaurosis
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30. Pharmacological approaches to retinitis pigmentosa: A laboratory perspective
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31. Lipid Nanoparticles for Ocular Gene Delivery
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32. Safety profile of recombinant adeno-associated viral vectors: focus on alipogene tiparvovec (Glybera ® )
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33. Recombinant Adeno-Associated Viral Vectors (rAAV)-Vector Elements in Ocular Gene Therapy Clinical Trials and Transgene Expression and Bioactivity Assays
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34. Special Issue Introduction: Inherited Retinal Disease: Novel Candidate Genes, Genotype–Phenotype Correlations, and Inheritance Models
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35. Dextran and Protamine-Based Solid Lipid Nanoparticles as Potential Vectors for the Treatment of X-Linked Juvenile Retinoschisis
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36. Potential of gene therapy as a treatment for heart failure
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37. A phase I trial of adeno-associated virus serotype 1-γ-sarcoglycan gene therapy for limb girdle muscular dystrophy type 2C
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39. Successful Amelioration of Mitochondrial Optic Neuropathy Using the Yeast NDI1 Gene in a Rat Animal Model
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40. Gene therapy for inherited retinal and optic nerve degenerations
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41. Clinical Perspectives and Trends: Microperimetry as a Trial Endpoint in Retinal Disease
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42. A Comprehensive Clinical and Biochemical Functional Study of a Novel RPE65 Hypomorphic Mutation
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49. Randomized trial of bilateral gene therapy injection for m.11778G&amp;gt;A <i>MT-ND4</i> Leber optic neuropathy
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