Antisense Oligonucleotide- and CRISPR-Cas9-Mediated Rescue of mRNA Splicing for a Deep Intronic CLRN1 Mutation
Mutations in CLRN1 cause Usher syndrome (USH) type III (USH3A), a disease characterized by progressive hearing impairment, retinitis pigmentosa, and vestibular dysfunction. Due to the lack of appropriate disease models, no efficient therapy for retinitis pigmentosa in USH patients exists so far. In...
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| Main Authors: | , , , , , , , , , , |
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| Format: | Book |
| Published: |
Elsevier,
2020-09-01T00:00:00Z.
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| Subjects: | |
| Online Access: | Connect to this object online. |
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