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Title | Creator | Description | Identifier |
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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 Sahel | | 20200310_nanos_sciplatform3_05 |
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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 Sahel | LHON 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 |
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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 Sahel | LHON 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 |