A protein domain-oriented approach to expand the opportunities of therapeutic exon skipping for USH2A-associated retinitis pigmentosa
Loss-of-function mutations in USH2A are among the most common causes of syndromic and non-syndromic retinitis pigmentosa (RP). We previously presented skipping of USH2A exon 13 as a promising treatment paradigm for USH2A-associated RP. However, RP-associated mutations are often private, and evenly d...
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| Main Authors: | , , , , , , , , |
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| Format: | Book |
| Published: |
Elsevier,
2023-06-01T00:00:00Z.
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| Subjects: | |
| Online Access: | Connect to this object online. |
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