Stargardt disease-associated in-frame ABCA4 exon 17 skipping results in significant ABCA4 function
Abstract Background ABCA4, the gene implicated in Stargardt disease (STGD1), contains 50 exons, of which 17 contain multiples of three nucleotides. The impact of in-frame exon skipping is yet to be determined. Antisense oligonucleotides (AONs) have been investigated in Usher syndrome-associated gene...
| Main Authors: | , , , , , , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
BMC
2023-08-01
|
| Series: | Journal of Translational Medicine |
| Online Access: | https://doi.org/10.1186/s12967-023-04406-x |