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Title | Creator | Description | Identifier |
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rAAV2/2-ND4 Treatment of Leber Optic Neuropathy: 72-Week Data from the REVERSE Phase III Clinical Trial | Nancy Newman, Mark Moster, Alfredo Sadun, Thomas Klopstock, Catherine Vignal, Valerio Carelli, Patrick Yu-Wai-Man, Jose Sahel, Barrett Katz | LHON 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 |
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rAAV2/2-ND4 Treatment of Leber Optic Neuropathy: 72-Week Data from the REVERSE Phase III Clinical Trial | Nancy Newman, Mark Moster, Alfredo Sadun, Thomas Klopstock, Catherine Vignal, Valerio Carelli, Patrick Yu-Wai-Man, Jose Sahel, Barrett Katz | LHON 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 |