Sang‐Yeon Lee

This paper introduces a method using virus-like particles to enable orthogonal genome editing by packaging distinct CRISPR effectors and gRNAs, allowing simultaneous gene editing without gRNA crosstalk in vitro and in vivo.

This study uses whole-genome sequencing and functional assays to improve genetic diagnosis of sensorineural hearing loss, achieving a 55.6% diagnostic yield through comprehensive genomic profiling.

This study uses base editing to correct a mutation causing hearing loss in a humanized mouse model, restoring hearing function and gene expression.

This study validates a whole-genome sequencing assay for rare disease genomic profiling, showing high sensitivity and positive predictive values for variant detection.

The paper develops CRISPR-based strategies to correct a complex genomic rearrangement in EYA1 linked to haploinsufficiency, showing successful gene expression restoration in patient-derived cells.

This study corrects a SSBP1 mutation using adenine base editors, improving mitochondrial function but with trade-offs between editing efficacy and off-target risks.

This study identifies TMEM43 mutations as a cause of autosomal dominant auditory neuropathy spectrum disorder by showing their interaction with Connexin-mediated channels in cochlear glia-like supporting cells.