Effective AAV-mediated gene replacement therapy in retinal organoids modeling AIPL1-associated LCA4
Biallelic variations in the aryl hydrocarbon receptor interacting protein-like 1 (AIPL1) gene cause Leber congenital amaurosis subtype 4 (LCA4), an autosomal recessive early-onset severe retinal dystrophy that leads to the rapid degeneration of retinal photoreceptors and the severe impairment of sig...
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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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