Development of all-in-one CRISPR/Cas9 and CRISPRi AAV constructs to treat autosomal dominant retinitis pigmentosa
<p>Dominant mutations in RHO (rhodopsin) are the most common cause of autosomal dominant retinitis pigmentosa (RHO-adRP). RHO-adRP causes progressive loss of rod cells, followed by cone cells, leading to blindness. This disease is a candidate for CRISPR gene editing, as reduction of mutant rho...
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| 格式: | Thesis |
| 语言: | English |
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2021
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