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TitleCreatorDescriptionIdentifier
1 Bilateral Visual Improvement with Unilateral Gene Therapy for Leber Hereditary Optic Neuropathy (LHON) (Video)Nancy J. Newman, Mark Moster, Alfredo Sadun, Thomas Klopstock, Catherine Vignal-Clermont, Valerio Carelli, Patrick Yu-Wai-Man, Robert Sergott, Valerie Biousse, Laure Blouin, Magali Taiel, Pierre Burguiere, Caroline Chevalier, Katz Barrett, Jose-Alain Sahel20200310_nanos_sciplatform3_05
2 Bilateral Visual Improvement with Unilateral Gene Therapy for Leber Hereditary Optic Neuropathy (LHON) (Slides)Nancy J. Newman, Mark Moster, Alfredo Sadun, Thomas Klopstock, Catherine Vignal-Clermont, Valerio Carelli, Patrick Yu-Wai-Man, Robert Sergott, Valerie Biousse, Laure Blouin, Magali Taiel, Pierre Burguiere, Caroline Chevalier, Katz Barrett, Jose-Alain SahelLHON is a mitochondrially-inherited disease causing severe bilateral optic neuropathies. A point mutation in the mitochondrial ND4-gene accounts for 75% of LHON cases. rAAV2/2-ND4 is a gene therapy e nabling allotopic expression and delivery of the wildtype ND4 protein to mitochondria of retinal gan...20200310_nanos_sciplatform3_05-slides
3 Bilateral Visual Improvement with Unilateral Gene Therapy for Leber Hereditary Optic Neuropathy (LHON) (PDF)Nancy J. Newman, Mark Moster, Alfredo Sadun, Thomas Klopstock, Catherine Vignal-Clermont, Valerio Carelli, Patrick Yu-Wai-Man, Robert Sergott, Valerie Biousse, Laure Blouin, Magali Taiel, Pierre Burguiere, Caroline Chevalier, Katz Barrett, Jose-Alain SahelLHON is a mitochondrially-inherited disease causing severe bilateral optic neuropathies. A point mutation in the mitochondrial ND4-gene accounts for 75% of LHON cases. rAAV2/2-ND4 is a gene therapy e nabling allotopic expression and delivery of the wildtype ND4 protein to mitochondria of retin...20200310_nanos_sciplatform3_05-pdf
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