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- [29] Antisense oligonucleotide therapy corrects splicing in the common Stargardt disease type 1-causing variant ABCA4 c.5461-10T>C MOLECULAR THERAPY NUCLEIC ACIDS, 2023, 31 : 674 - 688
- [30] Targeted next-generation sequencing determined a novel SGCG variant that is associated with limb-girdle muscular dystrophy type 2C: A case report CLINICAL CASE REPORTS, 2023, 11 (03):