Development of CRISPR gene therapy for retinal degenerations
<p>Inherited retinal degenerations (IRDs) are a leading cause of blindness, caused by mutations in genes leading to dysfunction and death of cells in the outer retina. Gene-supplementation therapies using adeno-associated viral (AAV) vectors have been developed, however, they are limited to th...
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フォーマット: | 学位論文 |
言語: | English |
出版事項: |
2021
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