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1 rAAV2/2-ND4 Treatment of Leber Optic Neuropathy: 72-Week Data from the REVERSE Phase III Clinical TrialNancy Newman, Mark Moster, Alfredo Sadun, Thomas Klopstock, Catherine Vignal, Valerio Carelli, Patrick Yu-Wai-Man, Jose Sahel, Barrett KatzLHON is a mitochondrially inherited disease that causes bilateral central vision loss. A point mutation in the mitochondrial ND4gene at nucleotide position 11778 accounts for 75% of LHON cases. rAAV2/2-ND4is a gene therapy enabling allotopic expression and delivery of the wildtype ND4 protein to mit...20190319_nanos_sciplatform3_01-video
2 rAAV2/2-ND4 Treatment of Leber Optic Neuropathy: 72-Week Data from the REVERSE Phase III Clinical TrialNancy Newman, Mark Moster, Alfredo Sadun, Thomas Klopstock, Catherine Vignal, Valerio Carelli, Patrick Yu-Wai-Man, Jose Sahel, Barrett KatzLHON is a mitochondrially inherited disease that causes bilateral central vision loss. A point mutation in the mitochondrial ND4gene at nucleotide position 11778 accounts for 75% of LHON cases. rAAV2/2-ND4is a gene therapy enabling allotopic expression and delivery of the wildtype ND4 protein to mit...20190319_nanos_sciplatform3_01-abstract
3 rAAV2/2-ND4 Treatment of Leber Optic Neuropathy: 72-Week Data from the REVERSE Phase III Clinical TrialNancy Newman, Mark Moster, Alfredo Sadun, Thomas Klopstock, Catherine Vignal, Valerio Carelli, Patrick Yu-Wai-Man, Jose Sahel, Barrett KatzLHON is a mitochondrially inherited disease that causes bilateral central vision loss. A point mutation in the mitochondrial ND4gene at nucleotide position 11778 accounts for 75% of LHON cases. rAAV2/2-ND4is a gene therapy enabling allotopic expression and delivery of the wildtype ND4 protein to mit...20190319_nanos_sciplatform3_01_slides
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