Single intravitreal administration of a tetravalent siRNA exhibits robust and efficient gene silencing in mouse and pig photoreceptors
Inherited retinal dystrophies caused by dominant mutations in photoreceptor (PR) cell expressed genes are a major cause of irreversible vision loss. Oligonucleotide therapy has been of interest in diseases that conventional medicine cannot target. In the early days, small interfering RNAs (siRNAs) w...
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| Main Authors: | , , , , , , , , , , , |
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| Format: | Book |
| Published: |
Elsevier,
2024-03-01T00:00:00Z.
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| Subjects: | |
| Online Access: | Connect to this object online. |
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