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1. Full Text Gene therapy for leber hereditary optic neuropathy initial results
by Feuer, William J and Schiffman, Joyce C and Davis, Janet L and Porciatti, Vittorio and Gonzalez, Phillip and Koilkonda, Rajeshwari D and Yuan, Huijun and Lalwani, Anil and Lam, Byron L and Guy, John
Ophthalmology (Rochester, Minn.), ISSN 0161-6420, 03/2016, Volume 123, Issue 3, pp. 558 - 570
Life Sciences & Biomedicine | Ophthalmology | Science & Technology | Electroretinography | Dependovirus - genetics | Genetic Therapy | Prospective Studies | Intravitreal Injections | Tomography, Optical Coherence | Humans | Middle Aged | Male | NADH Dehydrogenase - genetics | Optic Atrophy, Hereditary, Leber - genetics | Optic Atrophy, Hereditary, Leber - therapy | DNA, Mitochondrial - genetics | Visual Fields | Polymerase Chain Reaction | Adult | Female | Polymorphism, Single Nucleotide | Visual Field Tests | Genetic Vectors | Optic Atrophy, Hereditary, Leber - physiopathology | Visual Acuity - physiology | Genetic vectors | Gene therapy | Genes | Index Medicus
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2. Full Text Gene Therapy for Leber Hereditary Optic Neuropathy: Low- and Medium-Dose Visual Results
by Guy, John and Feuer, William J and Davis, Janet L and Porciatti, Vittorio and Gonzalez, Phillip J and Koilkonda, Rajeshwari D and Yuan, Huijun and Hauswirth, William W and Lam, Byron L
Ophthalmology (Rochester, Minn.), ISSN 0161-6420, 11/2017, Volume 124, Issue 11, pp. 1621 - 1634
Dependovirus - genetics | Genetic Therapy | Visual Fields - physiology | Prospective Studies | Follow-Up Studies | Intravitreal Injections | Humans | Middle Aged | Male | NADH Dehydrogenase - genetics | Retinal Ganglion Cells | Young Adult | DNA, Mitochondrial - genetics | Adult | Female | Optic Atrophy, Hereditary, Leber - physiopathology | Real-Time Polymerase Chain Reaction | Visual Acuity - physiology | Electroretinography | Tomography, Optical Coherence | Dependovirus - immunology | Optic Atrophy, Hereditary, Leber - therapy | Genetic Vectors | Nerve Fibers | Antibodies, Neutralizing - blood | Care and treatment | Genetic vectors | Gene therapy | Genes | Analysis | Blindness | Index Medicus
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3. Full Text Mitochondrial optic neuropathies – Disease mechanisms and therapeutic strategies
by Yu-Wai-Man, Patrick and Griffiths, Philip G and Chinnery, Patrick F
Progress in retinal and eye research, ISSN 1350-9462, 03/2011, Volume 30, Issue 2, pp. 81 - 114
Glaucoma | Hereditary spastic paraplegia | Dominant optic atrophy | Neuroprotection | Multiple sclerosis | Mitofusin | Optic neuropathy | Optic neuritis | Leber hereditary optic neuropathy | Mitochondrial DNA | Retinal ganglion cell | Life Sciences & Biomedicine | Ophthalmology | Science & Technology | Optic Nerve - pathology | Humans | Optic Atrophy, Hereditary, Leber - therapy | Retinal Ganglion Cells - pathology | Point Mutation | Optic Atrophy, Hereditary, Leber - pathology | Phenotype | Animals | DNA, Mitochondrial - genetics | Optic Atrophy, Autosomal Dominant - therapy | Optic Atrophy, Autosomal Dominant - pathology | Optic Atrophy, Autosomal Dominant - physiopathology | Optic Atrophy, Hereditary, Leber - physiopathology | Disease Models, Animal | Index Medicus
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4. Full Text Natural History of Conversion of Leber's Hereditary Optic Neuropathy: A Prospective Case Series
by Hwang, Tiffany Jean and Karanjia, Rustum and Moraes-Filho, Milton Nunes and Gale, Jesse and Tran, Jeffrey Show and Chu, Edward R and Salomao, Solange R and Berezovsky, Adriana and Belfort, Rubens and Moraes, Milton Nunes and Sadun, Federico and DeNegri, Anna Maria and La Morgia, Chiara and Barboni, Piero and Ramos, Carolina do V.F and Chicani, Carlos Filipe and Quiros, Peter A and Carelli, Valerio and Sadun, Alfredo A
Ophthalmology (Rochester, Minn.), ISSN 0161-6420, 06/2017, Volume 124, Issue 6, pp. 843 - 850
Life Sciences & Biomedicine | Ophthalmology | Science & Technology | Nerve Fibers - pathology | Visual Fields - physiology | Prospective Studies | Tomography, Optical Coherence | Humans | Middle Aged | Male | Optic Atrophy, Hereditary, Leber - genetics | Retinal Ganglion Cells - pathology | Young Adult | Vision Disorders - diagnosis | Vision Disorders - physiopathology | DNA, Mitochondrial - genetics | Pedigree | Adolescent | Adult | Female | Visual Field Tests | Optic Atrophy, Hereditary, Leber - diagnosis | Visual Acuity - physiology | Index Medicus
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5. Full Text Trial End Points and Natural History in Patients With G11778A Leber Hereditary Optic Neuropathy : Preparation for Gene Therapy Clinical Trial
by Lam, Byron L and Feuer, William J and Schiffman, Joyce C and Porciatti, Vittorio and Vandenbroucke, Ruth and Rosa, Potyra R and Gregori, Giovanni and Guy, John
JAMA ophthalmology, ISSN 2168-6165, 02/2014, Volume 132, Issue 4, pp. 428 - 436
Life Sciences & Biomedicine | Ophthalmology | Science & Technology | Dependovirus - genetics | Genetic Therapy | Visual Fields - physiology | Prospective Studies | Intravitreal Injections | Humans | Middle Aged | Male | NADH Dehydrogenase - genetics | Optic Atrophy, Hereditary, Leber - genetics | Retinal Ganglion Cells - metabolism | Retinal Ganglion Cells - pathology | Young Adult | DNA, Mitochondrial - genetics | DNA Mutational Analysis | Endpoint Determination | Adult | Female | Optic Atrophy, Hereditary, Leber - diagnosis | Child | Visual Acuity - physiology | Electroretinography | Nerve Fibers - pathology | Tomography, Optical Coherence | Clinical Trials as Topic | Optic Atrophy, Hereditary, Leber - therapy | Adolescent | Visual Field Tests | Genetic Vectors | Clinical trials | Care and treatment | Usage | Optic atrophy | Gene therapy | Medical history taking | Index Medicus | Abridged Index Medicus
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6. Full Text A randomized placebo-controlled trial of idebenone in Leber's hereditary optic neuropathy
by Klopstock, Thomas and Yu-Wai-Man, Patrick and Dimitriadis, Konstantinos and Rouleau, Jacinthe and Heck, Suzette and Bailie, Maura and Atawan, Alaa and Chattopadhyay, Sandip and Schubert, Marion and Garip, Aylin and Kernt, Marcus and Petraki, Diana and Rummey, Christian and Leinonen, Mika and Metz, Günther and Griffiths, Philip G and Meier, Thomas and Chinnery, Patrick F
Brain (London, England : 1878), ISSN 0006-8950, 9/2011, Volume 134, Issue 9, pp. 2677 - 2686
mitochondrial encephalomyopathy | optic atrophy | mitochondrial DNA | LHON | optic neuropathy | mitochondrial disease | idebenone | Neurosciences | Clinical Neurology | Neurosciences & Neurology | Life Sciences & Biomedicine | Science & Technology | Cranial nerves. Spinal roots. Peripheral nerves. Autonomic nervous system. Gustation. Olfaction | Neurology | Biological and medical sciences | Medical sciences | Nervous system (semeiology, syndromes) | Prospective Studies | Double-Blind Method | Humans | Middle Aged | Ubiquinone - analogs & derivatives | Male | Antioxidants - pharmacology | Ubiquinone - pharmacology | Young Adult | Optic Atrophy, Hereditary, Leber - drug therapy | Antioxidants - therapeutic use | Visual Acuity - drug effects | DNA, Mitochondrial - genetics | Retina - ultrastructure | Adolescent | Contrast Sensitivity - drug effects | Placebos | Ubiquinone - therapeutic use | Adult | Female | Aged | Mutation | Optic Atrophy, Hereditary, Leber - physiopathology | Index Medicus | Abridged Index Medicus | Original
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