Sangsu Bae

This paper introduces a gene replacement tool called Prime Assembly (PA) that efficiently and accurately replaces large DNA fragments in human cells, restoring gene function in disease-related genes.

This study shows that base editing in human embryos produces efficient on-target changes without chromosomal abnormalities or large deletions, unlike CRISPR/Cas9-induced DSBs.

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.

The paper introduces a nanopore sequencing method with Cas9 targeting and an analysis algorithm to detect STR expansions, identifying pathogenic repeats in cerebellar ataxia patients.

The paper uses AI to generate a small MLH1 binder that enhances prime editing efficiency by disrupting MMR, resulting in the highest PE efficiency to date.

This paper introduces snuABE, a single-nucleotide resolution ABE that reduces bystander effects through engineered ADAR and tagRNA design, achieving precise A-to-G editing in human cells.

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

The paper introduces CHEETAH, a system using reprogrammed tracrRNAs to capture and record intracellular RNAs in human cells, demonstrating its ability to self-edit, selectively record viral RNA fragments, and enable multiplexed, time-resolved recording.

The paper shows that bystander base editing with ABE8e impairs visual function restoration in LCA model mice by causing structural and functional disruptions in RPE65.

The paper identifies cellular dsRNA-binding proteins (dsRBPs) using anti-dsRNA immunoprecipitation and mass spectrometry, revealing their role in regulating the interferon response and antiviral defense against HCoV-OC43.

The paper identifies mechanisms by which CRISPR, base editors, and prime editors cause large DNA deletions and proposes strategies to mitigate them.

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

The paper identifies MSH2 and MSH6 as size-dependent determinants of prime editing efficiency in human embryonic stem cells, showing that their disruption significantly improves editing outcomes.

This paper tests prime editing in human pluripotent stem cells, showing it can induce various genetic modifications and correct disease mutations without off-target effects from the reverse transcriptase domain.

This study uses adenine base editing to correct a nonsense mutation in a mouse model of LCA, improving visual function.

The paper establishes isogenic GNE myopathy disease models using base editors in hPSCs, showing mutation-specific hyposialylation and drug responses.

CRISPR-pass uses adenine base editors to bypass nonsense mutations in patient-derived fibroblasts, showing potential for treating genetic disorders.

The paper develops a method to enrich genome-edited human pluripotent stem cells by inducing temporary drug resistance through SLC35F2 knockdown, achieving efficient selection in 3 weeks.

The paper introduces two web tools, BE-Designer and BE-Analyzer, for designing CRISPR base editor targets and analyzing next-generation sequencing data.

The paper questions the claim that unexpected SNVs and indels in CRISPR-treated mice are caused by CRISPR-Cas9, highlighting the lack of validation for these off-target effects.