Sequence-Specific Suppression of Alleles Causing Dominantly Inherited Retinal Degeneration Using the RNA-Guided Nuclease Cas9
Background: Dominant mutations account for a substantial proportion of inherited retinal degenerations (IRDs) through haploinsufficiency or gain-of-function (GOF) effects. This work explores a potential strategy to treat diseases caused by dominant GOF mutations by specifically suppressing the fun...
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| Format: | Others |
| Language: | en |
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Harvard University
2016
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| Online Access: | http://nrs.harvard.edu/urn-3:HUL.InstRepos:27007731 |